List of Orphan
Designationed Products Approved for Marketing
This list contains Orphan Designated products that have been approved for marketing. To search for a particular product, please use the Find option of your Web browser (generally this is found
under the Edit menu)
To open a copy of this list as an Excel Spreadsheet, click here Orphan Designated Products Approved for Marketing
Generic Name: Albendazole
| Trade Name: Albenza
|
Orphan Indication: Treatment of hydatid disease (cystic echinococcosis due to E. granulosus
larvae or alveolar echinococcosis due to E. multilocularis larvae).
Sponsor SmithKline Beecham Pharmaceuticals Contact Ms. Debra Hackett
Address Philadelphia, PA Phone: (215) 751-4000 Fax: (215) 751-3400
Status: Designated/Approved Designation 17-Jan-96 Marketing Approval 11-Jun-96
Generic Name: Albendazole
| Trade Name: Albenza
|
Orphan Indication: Treatment of neurocysticercosis due to Taenia solium as: 1) chemotherapy
of parenchymal, subarachnoidal and racemose (cysts in spinal fluid)
neurocysticercosis in symptomatic cases and 2) prophylaxis of epilepsy
and other sequelae in asymptomatic neurocysticercosis.
Sponsor SmithKline Beecham Pharmaceuticals Contact Ms. Debra Hackett
Address Philadelphia, PA Phone: (215) 751-4000 Fax: (215) 751-3400
Status: Designated/Approved Designation 18-Jan-96 Marketing Approval 11-Jun-96
Generic Name: Aldesleukin
| Trade Name: Proleukin
|
Orphan Indication: Treatment of metastatic melanoma.
Sponsor Chiron Corporation Contact Ms. Linda FitzGerald
Address Emeryville, CA Phone: 5109233575 Fax: (510) 923-3344
Status: Designated/Approved Designation 10-Sep-9 Marketing Approval 09-Jan-98
Generic Name: Aldesleukin
| Trade Name: Proleukin
|
Orphan Indication: Treatment of metastatic renal cell carcinoma.
Sponsor Chiron Corporation Contact Ms. Linda FitzGerald
Address Emeryville, CA Phone: 5109233575 Fax: (510) 923-3344
Status: Designated/Approved Designation 14-Sep-8 Marketing Approval 05-May-92
Generic Name: alemtuzumab
| Trade Name: Campath
|
Orphan Indication: Treatment of chronic lymphocytic leukemia.
Sponsor Millennium and ILEX Partners, LP Contact Mr. Mike Bernstein
Address Cambridge, MA Phone: 6175513618 Fax:
Status: Designated/Approved Designation 20-Oct-97 Marketing Approval 07-May-01
Generic Name: Alglucerase injection
| Trade Name: Ceredase
|
Orphan Indication: For replacement therapy in patients with Gaucher's disease type I.
Sponsor Genzyme Corporation Contact Ms. Suzanne Sensabaugh
Address Cambridge, MA Phone: (617) 374-7455 Fax: (617) 374-7470
Status: Designated/Approved Designation 11-Mar-85 Marketing Approval 05-Apr-91
Generic Name: Alitretinoin
| Trade Name: Panretin
|
Orphan Indication: Topical treatment of cutaneous lesions in patients with AIDS-related
Kaposi's sarcoma.
Sponsor Ligand Pharmaceuticals Inc. Contact Dr. David Furlano
Address San Diego, CA Phone: (619) 550-7600 Fax: (619) 550-1827
Status: Designated/Approved Designation 24-Mar-98 Marketing Approval 02-Feb-99
Generic Name: Allopurinol sodium
| Trade Name: Zyloprim for Injection
|
Orphan Indication: Management of patients with leukemia, lymphoma, and solid tumor
malignancies who are receiving cancer therapy which causes elevations of
serum and urinary uric acid levels and who cannot tolerate oral therapy.
Sponsor Catalytica Pharmaceuticals, Inc Contact Dr. Greg Little
Address Greenville, NC Phone: (800) 327-7106 Fax: ( ) -
Status: Designated/Approved Designation 16-Oct-92 Marketing Approval 17-May-96
Generic Name: Alpha1-proteinase inhibitor (human)
| Trade Name: Prolastin
|
Orphan Indication: For replacement therapy in the alpha-1-proteinase inhibitor congenital
deficiency state.
Sponsor Bayer Corporation Contact Dr. Brian Miller
Address New Haven, CT Phone: (800) 288-8371 Fax:
Status: Designated/Approved Designation 07-Dec-8 Marketing Approval 02-Dec-87
Generic Name: Altretamine
| Trade Name: Hexalen
|
Orphan Indication: Treatment of advanced adenocarcinoma of the ovary.
Sponsor Medimmune Oncology, Inc. Contact Ms. Eve Damiano
Address West Conshohocken, PA Phone: (610) 832-4580 Fax: (610) 832-4571
Status: Designated/Approved Designation 09-Feb-8 Marketing Approval 26-Dec-90
Generic Name: Amifostine
| Trade Name: Ethyol
|
Orphan Indication: For use as a chemoprotective agent for cisplatin in the treatment of
advanced ovarian carcinoma.
Sponsor Medimmune Oncology, Inc. Contact Ms. Eve Damiano
Address West Conshohocken, PA Phone: (610) 832-4580 Fax: 6108324671
Status: Designated/Approved Designation 30-May-9 Marketing Approval 08-Dec-95
0
Generic Name: Amifostine
| Trade Name: Ethyol
|
Orphan Indication: Reduction of the incidence of moderate to severe xerostomia in patients
undergoing post-operative radiation treatment for head and neck cancer.
Sponsor Medimmune Oncology, Inc. Contact Ms. Eve Damiano
Address Conshohocken, PA Phone: (610) 832-4580 Fax: (610) 832-4571
Status: Designated/Approved Designation 12-May-9 Marketing Approval 24-Jun-99
8
Generic Name: Aminosalicylic acid
| Trade Name: Paser Granules
|
Orphan Indication: Treatment of tuberculosis infections.
Sponsor Jacobus Pharmaceutical Company Contact Dr. Neil Lewis
Address Princeton, NJ Phone: (609) 921-7447 Fax:
Status: Designated/Approved Designation 19-Feb-9 Marketing Approval 30-Jun-94
Generic Name: Amiodarone HCl
| Trade Name: Cordarone
|
Orphan Indication: For the acute treatment and prophylaxis of life-threatening ventricular
tachycardia or ventricular fibrillation.
Sponsor Wyeth-Ayerst Laboratories Contact Dr. Eleanor DeLorme
Address Philadelphia, PA Phone: (215) 341-2239 Fax:
Status: Designated/Approved Designation 16-Mar-94 Marketing Approval 03-Aug-95
Generic Name: Amphotericin B lipid complex
| Trade Name: Abelcet
|
Orphan Indication: Treatment of invasive fungal infections.
Sponsor Liposome Company, Inc. Contact Dr. James Boyle
Address Princeton, NJ Phone: (609) 452-7060 Fax: (609) 452-1890
Status: Designated/Approved Designation 03-Dec-9 Marketing Approval 18-Oct-96
Generic Name: Anagrelide
| Trade Name: Agrylin
|
Orphan Indication: Treatment of essential thrombocythemia.
Sponsor Roberts Pharmaceutical Corp. Contact Mr. Drew Karlan
Address Eatontown, NJ Phone: (732) 389-1182 Fax: ( ) -
Status: Designated/Approved Designation 27-Jan-88 Marketing Approval 14-Mar-97
Generic Name: Antihemophilic factor (recombinant)
| Trade Name: ReFacto
|
Orphan Indication: For the control and prevention of hemorrhagic episodes and for surgical
prophylaxis in patients with hemophilia A (congenital factor VIII deficiency
or classic hemophilia).
Sponsor Genetics Institute, Inc. Contact Ms. Maryann Krane
Address Cambridge, MA Phone: (617) 665-8737 Fax: (617) 498-8838
Status: Designated/Approved Designation 08-Feb-9 Marketing Approval
Generic Name: Antihemophilic factor (recombinant)
| Trade Name: Kogenate
|
Orphan Indication: Prophylaxis and treatment of bleeding in individuals with hemophilia A or for
prophylaxis when surgery is required in individuals with hemophilia A.
Sponsor Bayer Corporation Contact Ms. Carol Moore
Address Elkhart, IN Phone: (510) 705-5224 Fax: (510) 705-5553
Status: Designated/Approved Designation 25-Sep-8 Marketing Approval 25-Feb-93
Generic Name: Antihemophilic factor/von Willebrand factor complex (human), dried,
| Trade Name: Humate-P
|
Orphan Indication: Treatment and prevention of bleeding in hemophilia A (classical hemophilia)
in adult patients; and treatment of spontaneous and trauma-induced
bleeding episodes in severe von Willebrand disease, and in mild and
moderate von Willebrand disease where use of desmopressin is known or
suspected to be inadequate in adult and pediatric patients.
Sponsor Aventis Behring L.L.C. Contact Ms. Carol Marchione
Address King Of Prussia, PA Phone: (610) 878-4026 Fax: (610) 878-4009
Status: Designated/Approved Designation 16-Oct-92 Marketing Approval 01-Apr-99
Generic Name: Antithrombin III (human)
| Trade Name: Thrombate III
|
Orphan Indication: For replacement therapy in congenital deficiency of AT-III for prevention
and treatment of thrombosis and pulmonary emboli.
Sponsor Bayer Corporation Contact Ms.
Address New Haven, CT Phone: (203) 937-2000 Fax:
Status: Designated/Approved Designation 26-Nov-8 Marketing Approval 30-Dec-91
Generic Name: Antithrombin III (human)
| Trade Name: ATnativ
|
Orphan Indication: For the treatment of patients with hereditary antithrombin III deficiency in
connection with surgical or obstetrical procedures or when they suffer from
thromboembolism.
Sponsor Pharmacia & Upjohn AB Contact Dr. Ronald Leonardi
Address Sweden, SE Phone: (858) 586-0751 Fax: ( ) -
Status: Designated/Approved Designation 08-Feb-8 Marketing Approval 13-Dec-89
Generic Name: Antivenin, crotalidae polyvalent immune Fab (ovine)
| Trade Name: CroFab
|
Orphan Indication: Treatment of envenomations inflicted by North American crotalid snakes.
Sponsor Protherics, Inc. Contact Ms. Carol Clark-Evans
Address Nashville, TN Phone: 6153271027 Fax: 6153201212
Status: Designated/Approved Designation 12-Jan-94 Marketing Approval 02-Oct-00
Generic Name: Aprotinin
| Trade Name: Trasylol
|
Orphan Indication: For prophylactic use to reduce perioperative blood loss and the homologous
blood transfusion requirement in patients undergoing cardiopulmonary
bypass surgery in the course of repeat coronary artery bypass graft
surgery, and in selected cases of primary coronary artery bypass graft
surgery where the risk of bleeding is especially high (impaired hemostasis)
or where transfusion is unavailable or unacceptable.
Sponsor Bayer Corporation Contact Dr. Christine Miller
Address West Haven, CT Phone: (203) 931-5145 Fax:
Status: Designated/Approved Designation 17-Nov-9 Marketing Approval 29-Dec-93
Generic Name: arsenic trioxide
| Trade Name: Trisenox
|
Orphan Indication: Treatment of myelodysplastic syndrome.
Sponsor Cell Therapeutics, Inc. Contact Ms. Jennie Allewell
Address Seattle, WA Phone: (206) 270-8424 Fax: (206) 270-8463
Status: Designated/Approved Designation 17-Jul-00 Marketing Approval 25-Sep-00
Generic Name: Arsenic trioxide
| Trade Name: Trisenox
|
Orphan Indication: Treatment of acute promyelocytic leukemia.
Sponsor Cell Therapeutics, Inc. Contact Ms. Jennie Allewell
Address Seattle, WA Phone: 2062708424 Fax: 2062708463
Status: Designated/Approved Designation 03-Mar-98 Marketing Approval 25-Sep-00
Generic Name: Atovaquone
| Trade Name: Mepron
|
Orphan Indication: Prevention of Pneumocystis carinii pneumonia (PCP) in high-risk,
HIV-infected patients defined by a history of one or more episodes of PCP
and/or a peripheral CD4+ (T4 helper/inducer) lymphocyte count less than or
Sponsor Glaxo Wellcome Research and Development Contact Mr. Thomas Shumaker
Address Research Triangle Park, NC Phone: (919) 483-9324 Fax: (919) 483-5756
Status: Designated/Approved Designation 14-Aug-9 Marketing Approval 05-Jan-99
Generic Name: Atovaquone
| Trade Name: Mepron
|
Orphan Indication: Treatment of AIDS associated Pneumocystis Carinii Pneumonia.
Sponsor Glaxo Wellcome Inc. Contact Dr. Michael Dalton
Address Research Triangle Park, NC Phone: (919) 483-2100 Fax:
Status: Designated/Approved Designation 10-Sep-9 Marketing Approval 25-Nov-92
Generic Name: Baclofen
| Trade Name: Lioresal Intrathecal
|
Orphan Indication: Treatment of intractable spasticity caused by spinal cord injury, multiple
sclerosis, and other spinal diseases (including spinal ischemia, spinal tumor,
transverse myelitis, cervical spondylosis, and degenerative myelopathy).
Sponsor Medtronic, Inc. Contact Mr. Darryle Peterson
Address Minneapolis, MN Phone: (612) 572-5000 Fax:
Status: Designated/Approved Designation 10-Nov-8 Marketing Approval 25-Jun-92
Generic Name: Basiliximab
| Trade Name: Simulect
|
Orphan Indication: Prophylaxis of solid organ rejection.
Sponsor Novartis Pharmaceuticals Corporation Contact Dr. Inmaculada Esparza
Address East Hanover, NJ Phone: (973) 781-3154 Fax: (973) 781-8364
Status: Designated/Approved Designation 12-Dec-9 Marketing Approval 12-May-98
Generic Name: Benzoate and phenylacetate
| Trade Name: Ucephan
|
Orphan Indication: For adjunctive therapy in the prevention and treatment of
hyperammonemia in patients with urea cycle enzymopathy due to
carbamylphosphate synthetase, ornithine, transcarbamylase, or
Sponsor B. Braun Medical Inc. Contact Mr. John D'Angelo
Address Irvine, CA Phone: (949) 660-2517 Fax:
Status: Designated/Approved Designation 21-Jan-86 Marketing Approval 23-Dec-87
Generic Name: Beractant
| Trade Name: Survanta intratracheal suspension
|
Orphan Indication: Treatment of neonatal respiratory distress syndrome.
Sponsor Ross Laboratories Contact Ms. Patricia Welch
Address Columbus, OH Phone: (614) 624-7283 Fax:
Status: Designated/Approved Designation 05-Feb-8 Marketing Approval 01-Jul-91
Generic Name: Beractant
| Trade Name: Survanta intratracheal suspension
|
Orphan Indication: Prevention of neonatal respiratory distress syndrome.
Sponsor Ross Laboratories Contact Ms. Patricia Welch
Address Columbus, OH Phone: (614) 624-7283 Fax:
Status: Designated/Approved Designation 05-Feb-8 Marketing Approval 01-Jul-91
Generic Name: Betaine
| Trade Name: Cystadane
|
Orphan Indication: Treatment of homocystinuria.
Sponsor Orphan Medical, Inc. Contact Dr. Dayton Reardan
Address Minnetonka, MN Phone: (612) 513-6900 Fax: (612) 541-9209
Status: Designated/Approved Designation 16-May-9 Marketing Approval 25-Oct-96
4
Generic Name: Bexarotene
| Trade Name: Targretin
|
Orphan Indication: Treatment of cutaneous manifestations of cutaneous T-cell lymphoma in
patients who are refractory to at least one prior systemic therapy.
Sponsor Ligand Pharmaceuticals, Inc. Contact Dr. Howard Holden
Address San Diego, CA Phone: (858) 550-7600 Fax: (858) 550-1827
Status: Designated/Approved Designation 18-Jun-99 Marketing Approval 29-Dec-99
Generic Name: Bleomycin sulfate
| Trade Name: Blenoxane
|
Orphan Indication: Treatment of malignant pleural effusion.
Sponsor Bristol-Myers Squibb Pharmaceutical Research Contact Dr. Joseph Sonk
Institute
Address Princeton, NJ Phone: (609) 252-5710 Fax:
Status: Designated/Approved Designation 17-Sep-9 Marketing Approval 20-Feb-96
Generic Name: Bosentan
| Trade Name: Tracleer
|
Orphan Indication: Treatment of pulmonary arterial hypertension.
Sponsor Actelion Life Sciences Ltd. Contact Ms. Jean Busch
Address San Mateo, CA Phone: (978) 887-0972 Fax: 9788877314
Status: Designated/Approved Designation 06-Oct-00 Marketing Approval 20-Nov-01
Generic Name: Botulinum toxin type A
| Trade Name: Botox
|
Orphan Indication: Treatment of blepharospasm associated with dystonia in adults (patients 12
years of age and above).
Sponsor Allergan, Inc. Contact Mr. Peter Kresel
Address Irvine, CA Phone: (714) 752-4500 Fax:
Status: Designated/Approved Designation 22-Mar-84 Marketing Approval 29-Dec-89
Generic Name: Botulinum toxin type A
| Trade Name: Botox
|
Orphan Indication: Treatment of strabismus associated with dystonia in adults (patients 12
years of age and above).
Sponsor Allergan, Inc. Contact Mr. Peter Kresel
Address Irvine, CA Phone: (714) 752-4500 Fax:
Status: Designated/Approved Designation 22-Mar-84 Marketing Approval 29-Dec-89
Generic Name: Botulinum toxin type A
| Trade Name: Botox
|
Orphan Indication: Treatment of cervical dystonia.
Sponsor Allergan, Inc. Contact Dr. Adelbert Stagg
Address Irvine, CA Phone: 7142466931 Fax:
Status: Designated/Approved Designation 20-Aug-8 Marketing Approval 21-Dec-00
Generic Name: Botulinum toxin type B
| Trade Name: NeuroBloc
|
Orphan Indication: Treatment of cervical dystonia.
Sponsor Elan Pharmaceuticals, Inc. Contact Mr. Steven Morrissey
Address South San Francisco, CA Phone: (800) 435-5108 Fax: (650) 616-5053
Status: Designated/Approved Designation 16-Jan-92 Marketing Approval 08-Dec-00
Generic Name: Buffered intrathecal electrolyte/dextrose injection
| Trade Name: Elliotts B Solution
|
Orphan Indication: For use as a diluent in the intrathecal administration of methotrexate and
cytarabine for the prevention or treatment meningeal leukemia and
lymphocytic lymphoma.
Sponsor Orphan Medical, Inc. Contact Dr. Dayton Reardon
Address Minnetonka, MN Phone: (612) 513-6900 Fax: (612) 541-9209
Status: Designated/Approved Designation 24-Aug-9 Marketing Approval 27-Sep-96
Generic Name: Busulfan
| Trade Name: Busulfex
|
Orphan Indication: As preparative therapy in the treatment of malignancies with bone marrow
transplantation.
Sponsor Orphan Medical, Inc. Contact Ms. Carol Curme
Address Minnetonka, MN Phone: 9525136974 Fax: 9525419209
Status: Designated/Approved Designation 28-Jul-94 Marketing Approval 04-Feb-99
Generic Name: Caffeine
| Trade Name: Cafcit
|
Orphan Indication: Treatment of apnea of prematurity.
Sponsor O.P.R. Development, L.P. Contact Mr. Sean Alan F.X. Reade
Address Lawrence, KS Phone: (614) 241-4131 Fax: (614) 276-0321
Status: Designated/Approved Designation 20-Sep-8 Marketing Approval 21-Sep-99
Generic Name: Calcitonin-human for injection
| Trade Name: Cibacalcin
|
Orphan Indication: Treatment of symptomatic Paget's disease (osteitis deformans).
Sponsor Novartis Pharmaceutical Corporation Contact Ms. Nancy Price
Address East Hanover, NJ Phone: (201) 503-7500 Fax:
Status: Designated/Approved Designation 20-Jan-87 Marketing Approval 31-Oct-86
Generic Name: Calcium acetate
| Trade Name: Phos-Lo
|
Orphan Indication: Treatment of hyperphosphatemia in end stage renal failure.
Sponsor Braintree Laboratories Contact Dr. Mark Cleveland
Address Braintree, MA Phone: (617) 843-2202 Fax:
Status: Designated/Approved Designation 22-Dec-8 Marketing Approval 10-Dec-90
Generic Name: Chenodiol
| Trade Name: Chenix
|
Orphan Indication: For patients with radiolucent stones in well opacifying gallbladders, in whom
elective surgery would be undertaken except for the presence of increased
surgical risk due to systemic disease or age.
Sponsor Solvay Contact Mr. George Severn
Address Marietta, GA Phone: (404) 578-9000 Fax:
Status: Designated/Approved Designation 21-Sep-8 Marketing Approval 28-Jul-83
Generic Name: Citric acid, glucono-delta-lactone and magnesium carbonate
| Trade Name: Renacidin Irrigation
|
Orphan Indication: Treatment of renal and bladder calculi of the apatite or struvite variety.
Sponsor United-Guardian, Inc. Contact Mr. Robert Rubinger
Address Smithtown, NY Phone: (516) 273-0900 Fax:
Status: Designated/Approved Designation 28-Aug-8 Marketing Approval 02-Oct-90
Generic Name: Cladribine
| Trade Name: Leustatin Injection
|
Orphan Indication: Treatment of hairy cell leukemia.
Sponsor R. W. Johnson Pharmaceutical Research Institute Contact Ms. Jacqueline Coelln
Address Raritan, NJ Phone: (908) 704-5829 Fax:
Status: Designated/Approved Designation 15-Nov-9 Marketing Approval 26-Feb-93
Generic Name: Clofazimine
| Trade Name: Lamprene
|
Orphan Indication: Treatment of lepromatous leprosy, including dapsone-resistant lepromatous
leprosy and lepromatous leprosy complicated by erythema nodosum
Sponsor Novartis Pharmaceutical Corporation Contact Ms. Nancy Price
Address East Hanover, NJ Phone: (201) 503-7500 Fax:
Status: Designated/Approved Designation 11-Jun-84 Marketing Approval 15-Dec-86
Generic Name: Clonidine
| Trade Name: Duraclon
|
Orphan Indication: For continous epidural administration as adjunctive therapy with intraspinal
opiates for the treatment of pain in cancer patients tolerant to, or
unresponsive to, intraspinal opiates.
Sponsor Roxane Laboratories, Inc. Contact Mr. Sean Alan F.X. Reade
Address Columbus, OH Phone: (614) 241-4131 Fax: (614) 276-0321
Status: Designated/Approved Designation 24-Jan-89 Marketing Approval 02-Oct-96
Generic Name: Coagulation factor IX
| Trade Name: Mononine
|
Orphan Indication: Replacement treatment and prophylaxis of the hemorrhagic complications
of hemophilia B.
Sponsor Armour Pharmaceutical Company Contact Mr. Stewart Mueller
Address Collegeville, PA Phone: (215) 540-8139 Fax:
Status: Designated/Approved Designation 27-Jun-89 Marketing Approval 20-Aug-92
Generic Name: Coagulation Factor IX (human)
| Trade Name: AlphaNine
|
Orphan Indication: For use as replacement therapy in patients with hemophilia B for the
prevention and control of bleeding episodes, and during surgery to correct
defective hemostasis.
Sponsor Alpha Therapeutic Corporation Contact Ms. M. Sue Preston
Address Los Angeles, CA Phone: (213) 225-2221 Fax:
Status: Designated/Approved Designation 05-Jul-90 Marketing Approval 31-Dec-90
Generic Name: Coagulation Factor IX (recombinant)
| Trade Name: BeneFix
|
Orphan Indication: Treatment of hemophilia B.
Sponsor Genetics Institute, Inc. Contact Dr. Frederick Gates
Address Cambridge, MA Phone: (617) 503-7332 Fax:
Status: Designated/Approved Designation 03-Oct-94 Marketing Approval 11-Feb-97
Generic Name: Coagulation factor VIIa (recombinant)
| Trade Name: NovoSeven
|
Orphan Indication: Treatment of bleeding episodes in hemophilia A or B patients with inhibitors
to Factor VIII or Factor IX.
Sponsor Novo Nordisk Pharmaceuticals, Inc. Contact Dr. Barry Reit
Address Princeton, NJ Phone: (609) 987-5877 Fax: (609) 921-8082
Status: Designated/Approved Designation 06-Jun-88 Marketing Approval 25-Mar-99
Generic Name: Colfosceril palmitate, cetyl alcohol, tyloxapol
| Trade Name: Exosurf Neonatal for Intratracheal Suspension
|
Orphan Indication: Prevention of hyaline membrane disease, also known as respiratory
distress syndrome, in infants born at 32 weeks gestation or less.
Sponsor Glaxo Wellcome Inc. Contact Dr. Michael Dalton
Address Research Triangle Park, NC Phone: (919) 483-2100 Fax:
Status: Designated/Approved Designation 20-Oct-89 Marketing Approval 02-Aug-90
Generic Name: Colfosceril palmitate, cetyl alcohol, tyloxapol
| Trade Name: Exosurf Neonatal for Intratracheal Suspension
|
Orphan Indication: Treatment of established hyaline membrane disease at all gestational ages.
Sponsor Glaxo Wellcome Inc. Contact Dr. Michael Dalton
Address Research Triangle Park, NC Phone: (919) 483-2100 Fax:
Status: Designated/Approved Designation 20-Oct-89 Marketing Approval 02-Aug-90
Generic Name: Corticorelin ovine triflutate
| Trade Name: Acthrel
|
Orphan Indication: For use in differentiating pituitary and ectopic production of ACTH in
patients with ACTH-dependent Cushings syndrome.
Sponsor Ferring Laboratories, Inc. Contact Mr. Isidoro Nudelman
Address Suffern, NY Phone: (888) 793-6367 Fax: ( ) -
Status: Designated/Approved Designation 24-Nov-8 Marketing Approval 23-May-96
Generic Name: Cromolyn sodium
| Trade Name: Gastrocrom
|
Orphan Indication: Treatment of mastocytosis.
Sponsor Fisons Corporation Contact Ms. Susan Raymond
Address Rochester, NY Phone: (716) 475-9000 Fax:
Status: Designated/Approved Designation 08-Mar-84 Marketing Approval 22-Dec-89
Generic Name: Cromolyn sodium 4% ophthalmic solution
| Trade Name: Opticrom 4% ophthalmic solution
|
Orphan Indication: Treatment of vernal keratoconjunctivitis.
Sponsor Fisons Corporation Contact Ms. Susan Raymond
Address Rochester, NY Phone: (716) 475-9000 Fax:
Status: Designated/Approved Designation 24-Jul-85 Marketing Approval 03-Oct-84
Generic Name: Cysteamine
| Trade Name: Cystagon
|
Orphan Indication: Treatment of nephropathic cystinosis.
Sponsor Mylan Laboratories, Inc. Contact Dr. John O'Donnell
Address Morgantown, WV Phone: (304) 599-2595 Fax:
Status: Designated/Approved Designation 25-Jan-91 Marketing Approval 15-Aug-94
Generic Name: Cytarabine liposomal
| Trade Name: DepoCyt
|
Orphan Indication: Treatment of neoplastic meningitis.
Sponsor DepoTech Corporation Contact Mr. Raymond Lamy
Address San Diego, CA Phone: (619) 625-2414 Fax: (619) 558-6617
Status: Designated/Approved Designation 02-Jun-93 Marketing Approval 01-Apr-99
Generic Name: Cytomegalovirus immune globulin (human)
| Trade Name: CytoGam
|
Orphan Indication: Prevention or attenuation of primary cytomegalovirus disease in
immunosuppressed recipients of organ transplants.
Sponsor Massachussetts Public Health Biologic Contact Dr. Jeanne Leszczynski
Address Boston, MA Phone: (617) 983-6400 Fax:
Status: Designated/Approved Designation 03-Aug-8 Marketing Approval 04-Dec-98
Generic Name: Daclizumab
| Trade Name: Zenapax
|
Orphan Indication: Prevention of acute renal allograft rejection.
Sponsor Hoffmann-La Roche, Inc. Contact Ms. Ileana Leon Brown
Address Nutley, NJ Phone: (201) 812-3720 Fax: (201) 812-3700
Status: Designated/Approved Designation 05-Mar-93 Marketing Approval 10-Dec-97
Generic Name: Daunorubicin citrate liposome injection
| Trade Name: DaunoXome
|
Orphan Indication: Treatment of patients with advanced HIV-associated Kaposi's sarcoma.
Sponsor NeXstar Pharmaceuticals, Inc. Contact Mr. Stephen Campbell
Address San Dimas, CA Phone: (909) 592-8530 Fax:
Status: Designated/Approved Designation 14-May-9 Marketing Approval 08-Apr-96
3
Generic Name: Denileukin diftitox
| Trade Name: Ontak
|
Orphan Indication: Treatment of patients with persistent or recurrent cutaneous T-cell
lymphoma whose malignant cells express the CD25 component of the IL-2
Sponsor Seragen, Inc. Contact Dr. Jill Hillier
Address Hopkinton, MA Phone: (508) 435-2331 Fax: (508) 435-0558
Status: Designated/Approved Designation 21-Aug-9 Marketing Approval 05-Feb-99
Generic Name: Desmopressin acetate
Trade Name:
|
Orphan Indication: Treatment of mild hemophilia A and von Willebrand's disease.
Sponsor Aventis Behring L.L.C. Contact Ms. Carol Marchione
Address King of Prussia, PA Phone: 6108784026 Fax:
Status: Designated/Approved Designation 22-Jan-91 Marketing Approval 07-Mar-94
Generic Name: Dexrazoxane
| Trade Name: Zinecard
|
Orphan Indication: For the prevention of cardiomyopathy associated with doxorubicin
administration.
Sponsor Pharmacia & Upjohn Contact Mr. Robert Paarlberg
Address Kalamazoo, MI Phone: (616) 833-0646 Fax: (616) 833-8237
Status: Designated/Approved Designation 17-Dec-9 Marketing Approval 26-May-95
Generic Name: Diazepam viscous solution for rectal administration
Trade Name:
|
Orphan Indication: For the management of selected, refractory, patients with epilepsy, on
stable regimens of antiepileptic drugs (AEDs), who require intermittent use
of diazepam to control bouts of increased seizure activity.
Sponsor Athena Neurosciences, Inc. Contact Ms. Kathleen Dumas
Address South San Francisco, CA Phone: (415) 877-0900 Fax: (415) 877-8370
Status: Designated/Approved Designation 25-Feb-9 Marketing Approval 29-Jul-97
Generic Name: Diethyldithiocarbamate
| Trade Name: Imuthiol
|
Orphan Indication: Treatment of AIDS.
Sponsor Connaught Laboratories Contact Dr. Pinya Cohen
Address Swiftwater, PA Phone: (717) 839-4590 Fax:
Status: Designated/Approved Designation 03-Apr-86 Marketing Approval
Generic Name: Digoxin immune FAB (Ovine)
| Trade Name: Digibind
|
Orphan Indication: Treatment of potentially life threatening digitalis intoxication in patients who
are refractory to management by conventional therapy.
Sponsor Glaxo Wellcome Inc. Contact Dr. Michael Dalton
Address Research Triangle Park, NC Phone: (919) 483-2100 Fax:
Status: Designated/Approved Designation 01-Nov-8 Marketing Approval 22-Apr-86
Generic Name: Dornase alfa
| Trade Name: Pulmozyme
|
Orphan Indication: To reduce mucous viscosity and enable the clearance of airway secretions
in patients with cystic fibrosis.
Sponsor Genentech, Inc. Contact Dr. M. David MacFarlane
Address South San Francisco, CA Phone: (415) 225-1000 Fax: (415) 225-6000
Status: Designated/Approved Designation 16-Jan-91 Marketing Approval 30-Dec-93
Generic Name: Doxorubicin liposome
| Trade Name: Doxil
|
Orphan Indication: Treatment of ovarian cancer.
Sponsor Alza Corporation Contact Mr. Thomas Tarlow
Address Mountain View, CA Phone: (650) 494-5000 Fax: (650) 321-2417
Status: Designated/Approved Designation 04-Nov-9 Marketing Approval 28-Jun-99
Generic Name: Dronabinol
| Trade Name: Marinol
|
Orphan Indication: For the stimulation of appetite and prevention of weight loss in patients with
a confirmed diagnosis of AIDS.
Sponsor Unimed Pharmaceuticals, Inc. Contact Dr. Margaret Hurley
Address Buffalo Grove, IL Phone: 9737011596 Fax: 9736359881
Status: Designated/Approved Designation 15-Jan-91 Marketing Approval 22-Dec-92
Generic Name: Eflornithine HCl
| Trade Name: Ornidyl
|
Orphan Indication: Treatment of Trypanosoma brucei gambiense infection (sleeping sickness).
Sponsor Hoechst Marion Roussel Contact Dr. Jack Dunn
Address Kansas City, MO Phone: (816) 966-5000 Fax:
Status: Designated/Approved Designation 23-Apr-86 Marketing Approval 28-Nov-90
Generic Name: Epirubicin
| Trade Name: Ellence
|
Orphan Indication: Treatment of breast cancer.
Sponsor Pharmacia & Upjohn Company Contact Ms. Denise Tindle
Address Kalamazoo, MI Phone: (616) 833-3825 Fax: (616) 833-8237
Status: Designated/Approved Designation 14-Sep-9 Marketing Approval 15-Sep-99
Generic Name: Epoetin alfa
| Trade Name: Epogen
|
Orphan Indication: Treatment of anemia associated with end stage renal disease.
Sponsor Amgen, Inc. Contact Mr. Ralph Smalling
Address Thousand Oaks, CA Phone: (805) 447-1000 Fax: (805) 499-8670
Status: Designated/Approved Designation 10-Apr-86 Marketing Approval 01-Jun-89
Generic Name: Epoetin alfa
| Trade Name: Epogen
|
Orphan Indication: Treatment of anemia associated with HIV infection or HIV treatment.
Sponsor Amgen, Inc. Contact Mr. Ralph Smalling
Address Thousand Oaks, CA Phone: (805) 447-1000 Fax: (805) 499-8670
Status: Designated/Approved Designation 01-Jul-91 Marketing Approval 31-Dec-90
Generic Name: Epoprostenol
| Trade Name: Flolan
|
Orphan Indication: Treatment of secondary pulmonary hypertension due to intrinsic
precapillary pulmonary vascular disease.
Sponsor GlaxoSmithKline Contact Mr. Roger Gaby
Address Research Triangle Park, NC Phone: (919) 483-9035 Fax:
Status: Designated/Approved Designation 22-Mar-99 Marketing Approval 14-Apr-00
Generic Name: Epoprostenol
| Trade Name: Flolan
|
Orphan Indication: Treatment of primary pulmonary hypertension.
Sponsor Glaxo Wellcome Inc. Contact Mr. Roger Gaby
Address Research Triangle Park, NC Phone: (919) 483-9035 Fax: (919) 315-0033
Status: Designated/Approved Designation 25-Sep-8 Marketing Approval 20-Sep-95
Generic Name: Etanercept
| Trade Name: Enbrel
|
Orphan Indication: Reduction in signs and symptoms of moderately to severely active
polyarticular-course juvenile rheumatoid arthritis in patients who have had
an inadequate response to one or more disease-modifying anti-rheumatic
Sponsor Immunex Corporation Contact Ms. Sally Gould
Address Seattle, WA Phone: (206) 389-4097 Fax:
Status: Designated/Approved Designation 27-Oct-98 Marketing Approval 27-May-99
Generic Name: Ethanolamine oleate
| Trade Name: Ethamolin
|
Orphan Indication: Treatment of patients with esophageal varices that have recently bled, to
prevent rebleeding.
Sponsor Block Drug Company, Inc. Contact Dr. Richard Bourne
Address Jersey City, NJ Phone: (201) 434-3000 Fax:
Status: Designated/Approved Designation 22-Mar-84 Marketing Approval 22-Dec-88
Generic Name: Etidronate disodium
| Trade Name: Didronel
|
Orphan Indication: Treatment of hypercalcemia of malignancy inadequately managed by
dietary modification and/or oral hydration.
Sponsor MGI Pharma, Inc. Contact Dr. Jo Gustafson
Address Bloomington, MN Phone: (612) 346-4722 Fax: (612) 346-4800
Status: Designated/Approved Designation 21-Mar-86 Marketing Approval 21-Apr-87
Generic Name: Exemestane
| Trade Name: Aromasin
|
Orphan Indication: Treatment of advanced breast cancer in postmenopausal women whose
disease has progressed following tamoxifen therapy.
Sponsor Pharmacia & Upjohn Contact Ms. Cecilia Blomqvist
Address Kalamazoo, MI Phone: (616) 833-0774 Fax: (616) 833-8237
Status: Designated/Approved Designation 19-Sep-9 Marketing Approval 21-Oct-99
Generic Name: Felbamate
| Trade Name: Felbatol
|
Orphan Indication: Treatment of Lennox-Gastaut syndrome.
Sponsor Wallace Laboratories Contact Dr. Monroe Klein
Address Princeton, NJ Phone: (609) 951-2079 Fax:
Status: Designated/Approved Designation 24-Jan-89 Marketing Approval 29-Jul-93
Generic Name: Filgrastim
| Trade Name: Neupogen
|
Orphan Indication: Reduction in the duration of neutropenia, fever, antibiotic use, and
hospitalization, following induction and consolidation treatment for acute
Sponsor Amgen, Inc. Contact Mr. Jeff Fellows
Address Thousand Oaks, CA Phone: (805) 447-8972 Fax: (805) 498-9377
Status: Designated/Approved Designation 07-Nov-9 Marketing Approval 02-Apr-98
Generic Name: Filgrastim
| Trade Name: Neupogen
|
Orphan Indication: Treatment of neutropenia associated with bone marrow transplants.
Sponsor Amgen, Inc. Contact Mr. Ralph Smalling
Address Thousand Oaks, CA Phone: (805) 447-1000 Fax: (805) 499-8670
Status: Designated/Approved Designation 01-Oct-90 Marketing Approval 15-Jun-94
Generic Name: Filgrastim
| Trade Name: Neupogen
|
Orphan Indication: Treatment of patients with severe chronic neutropenia (absolute neutrophil
count less than 500/mm3).
Sponsor Amgen, Inc. Contact Mr. Ralph Smalling
Address Thousand Oaks, CA Phone: (805) 447-1000 Fax: (805) 499-8670
Status: Designated/Approved Designation 07-Nov-9 Marketing Approval 19-Dec-94
Generic Name: Filgrastim
| Trade Name: Neupogen
|
Orphan Indication: For use in the mobilization of peripheral blood progenitor cells for collection
in patients who will receive myeloablative or myelosuppressive
Sponsor Amgen, Inc. Contact Mr. Ralph Smalling
Address Thousand Oaks, CA Phone: (805) 447-1000 Fax: (805) 499-8670
Status: Designated/Approved Designation 17-Jul-95 Marketing Approval 28-Dec-95
Generic Name: Fludarabine phosphate
| Trade Name: Fludara
|
Orphan Indication: Treatment of chronic lymphocytic leukemia (CLL), including refractory CLL.
Sponsor Berlex Laboratories, Inc. Contact Mr. Anthony Bourdakis
Address Richmond, CA Phone: (510) 262-5000 Fax: (510) 262-7053
Status: Designated/Approved Designation 18-Apr-89 Marketing Approval 18-Apr-91
Generic Name: Follitropin alfa, recombinant
| Trade Name: Gonal-F
|
Orphan Indication: For the initiation and re-initiation of spermatogenesis in adult males with
reproductive failure due to hypothalamic or pituitary dysfunction,
hypogonadotropic hypogonadism. AMENDED indication 6/27/00: For the
induction of spermatogenesis in men with primary and secondary
hypogonadotropic hypogonadism in whom the cause of infertility is not due
to primary testicular failure.
Sponsor Serono Laboratories, Inc. Contact Ms. Pamela Williamson
Joyce
Address Norwell, MA Phone: 7816812298 Fax: (781) 871-6754
Status: Designated/Approved Designation 21-Dec-9 Marketing Approval 24-May-00
Generic Name: Fomepizole
| Trade Name: Antizole
|
Orphan Indication: Treatment of methanol or ethylene glycol poisoning.
Sponsor Orphan Medical, Inc. Contact Ms. Carol Curme
Address Minnetonka, MN Phone: 9525136974 Fax: 9525419209
Status: Designated/Approved Designation 22-Dec-8 Marketing Approval 04-Dec-97
Generic Name: Fomepizole
| Trade Name: Antizole
|
Orphan Indication: Treatment of methanol or ethylene glycol poisoning.
Sponsor Orphan Medical, Inc. Contact Ms. Carol Curme
Address Minnetonka, MN Phone: 9525136974 Fax: 9525419209
Status: Designated/Approved Designation 22-Dec-8 Marketing Approval 08-Dec-00
Generic Name: Fosphenytoin
| Trade Name: Cerebyx
|
Orphan Indication: For the acute treatment of patients with status epilepticus of the grand mal
type.
Sponsor Warner-Lambert Company Contact Ms. Janeth Turner
Address Ann Arbor, MI Phone: (313) 996-7000 Fax: (313) 998-3283
Status: Designated/Approved Designation 04-Jun-91 Marketing Approval 05-Aug-96
Generic Name: Gallium nitrate injection
| Trade Name: Ganite
|
Orphan Indication: Treatment of hypercalcemia of malignancy.
Sponsor Solopak Pharmaceutical Co. Contact Mr. Jeffrey Ferguson
Address Elk Grove Village, IL Phone: (847) 806-0080 Fax:
Status: Designated/Approved/Wi Designation 05-Dec-8 Marketing Approval 17-Jan-91
thdrawn
Generic Name: Ganciclovir intravitreal implant
| Trade Name: Vitrasert Implant
|
Orphan Indication: Treatment of cytomegalovirus retinitis.
Sponsor Bausch & Lomb Surgical, Chiron Vision Products Contact Dr. Judy Gordon
Address Irvine, CA Phone: (714) 768-4690 Fax: (714) 468-4882
Status: Designated/Approved Designation 07-Jun-95 Marketing Approval 04-Mar-96
Generic Name: Ganciclovir sodium
| Trade Name: Cytovene
|
Orphan Indication: Treatment of cytomegalovirus retinitis in immunocompromised patients with
AIDS.
Sponsor Syntex (USA), Inc. Contact Michael Perry
Address Palo Alto, CA Phone: (415) 354-7453 Fax:
Status: Designated/Approved/Wi Designation 31-Oct-85 Marketing Approval 23-Jun-89
thdrawn
Generic Name: Gemtuzumab zogamicin
| Trade Name: Mylotarg
|
Orphan Indication: Treatment of CD33-positive acute myeloid leukemia.
Sponsor Wyeth-Ayerst Laboratories Contact Mr. Barry Sickels
Address Philadelphia, PA Phone: (610) 902-3742 Fax: (610) 964-5973
Status: Designated/Approved Designation 24-Nov-9 Marketing Approval 17-May-00
Generic Name: Glatiramer acetate
| Trade Name: Copaxone
|
Orphan Indication: Treatment of multiple sclerosis.
Sponsor Teva Pharmaceuticals USA Contact Dr. Stanley Scheindlin
Address Kulpsville, PA Phone: (800) 999-8382 Fax:
Status: Designated/Approved Designation 09-Nov-8 Marketing Approval 20-Dec-96
Generic Name: Gonadorelin acetate
| Trade Name: Lutrepulse
|
Orphan Indication: For induction of ovulation in women with hypothalamic amenorrhea due to a
deficiency or absence in the quantity or pulse pattern of endogenous
GnRH secretion.
Sponsor Ferring Laboratories, Inc. Contact Mr. Isidoro Nudelman
Address Suffern, NY Phone: (888) 793-6367 Fax:
Status: Designated/Approved Designation 22-Apr-87 Marketing Approval 10-Oct-89
Generic Name: Halofantrine
| Trade Name: Halfan
|
Orphan Indication: Treatment of mild to moderate acute malaria caused by susceptible strains
of P. falciparum and P. vivax.
Sponsor SmithKline Beecham Pharmaceuticals Contact Dr. Paula Goldberg
Address Philadelphia, PA Phone: (215) 751-4000 Fax: (215) 751-3400
Status: Designated/Approved Designation 04-Nov-9 Marketing Approval 24-Jul-92
Generic Name: Hemin
| Trade Name: Panhematin
|
Orphan Indication: Amelioration of recurrent attacks of acute intermittent porphyria (AIP)
temporarily related to the menstrual cycle in susceptible women and similar
symptoms which occur in other patients with AIP, porphyria variegata and
hereditary coproporphyria.
Sponsor Abbott Laboratories Contact Medical Information
Address Abbott Park, IL Phone: 8006339110 Fax:
Status: Designated/Approved Designation 16-Mar-84 Marketing Approval 20-Jul-83
Generic Name: Histrelin acetate
| Trade Name: Supprelin Injection
|
Orphan Indication: Treatment of central precocious puberty.
Sponsor Roberts Pharmaceutical Corp. Contact Mr. Drew Karlan
Address Eatontown, NJ Phone: (732) 389-1182 Fax:
Status: Designated/Approved Designation 10-Aug-8 Marketing Approval 24-Dec-91
Generic Name: Hydroxyurea
| Trade Name: Droxia
|
Orphan Indication: Treatment of patients with sickle cell anemia as shown by the presence of
hemoglobin S.
Sponsor Bristol-Myers Squibb Pharmaceutical Research Contact Dr. Joseph Linkewich
Institute
Address Princeton, NJ Phone: (609) 252-5761 Fax:
Status: Designated/Approved Designation 01-Oct-90 Marketing Approval 25-Feb-98
Generic Name: Idarubicin HCl for injection
| Trade Name: Idamycin
|
Orphan Indication: Treatment of acute myelogenous leukemia, also referred to as acute
nonlymphocytic leukemia.
Sponsor Adria Laboratories, Inc. Contact Mr. Douglas Jones
Address Columbus, OH Phone: (614) 764-8100 Fax: (614) 764-8102
Status: Designated/Approved Designation 25-Jul-88 Marketing Approval 27-Sep-90
Generic Name: Ifosfamide
| Trade Name: Ifex
|
Orphan Indication: Treatment of testicular cancer.
Sponsor Bristol-Myers Squibb Pharmaceutical Research Contact Dr. Douglas Kriesel
Institute
Address Wallingford, CT Phone: (203) 284-6051 Fax:
Status: Designated/Approved Designation 20-Jan-87 Marketing Approval 30-Dec-88
Generic Name: Imatinib
| Trade Name: Gleevec
|
Orphan Indication: Treatment of chronic myelogenous leukemia
Sponsor Novartis Pharmaceuticals Corporation Contact Ms. Ellen Cutler
Address East Hanover, NJ Phone: 9737818180 Fax: 9737816325
Status: Designated/Approved Designation 31-Jan-01 Marketing Approval 10-May-01
Generic Name: Imiglucerase
| Trade Name: Cerezyme
|
Orphan Indication: Replacement therapy in patients with types I, II, and III Gaucher's disease.
Sponsor Genzyme Corporation Contact Ms. Suzanne Sensabaugh
Address Cambridge, MA Phone: (617) 374-7455 Fax: (617) 374-7470
Status: Designated/Approved Designation 05-Nov-9 Marketing Approval 23-May-94
Generic Name: Immune globulin intravenous, human
| Trade Name: Gamimune N
|
Orphan Indication: Infection prophylaxis in pediatric patients affected with the human
immunodeficiency virus.
Sponsor Bayer Corporation Contact Ms.
Address New Haven, CT Phone: (203) 937-2000 Fax:
Status: Designated/Approved Designation 18-Feb-9 Marketing Approval 27-Dec-93
Generic Name: Infliximab
| Trade Name: Remicade
|
Orphan Indication: Treatment of moderately to severely active Crohn's disease for the
reduction of the signs and symptoms, in patients who have an inadequate
response to conventional therapy; and treatment of patients with fistulizing
Crohn's disease for the reduction in the number of draining enterocutaneous
Sponsor Centocor, Inc. Contact Ms. Wan Yin Jung
Address Malvern, PA Phone: (610) 889-4523 Fax: (610) 651-6123
Status: Designated/Approved Designation 14-Nov-9 Marketing Approval 24-Aug-98
Generic Name: Interferon alfa-2a
| Trade Name: Roferon A
|
Orphan Indication: Treatment of chronic myelogenous leukemia.
Sponsor Hoffmann-La Roche, Inc. Contact Ms. Loni da Silva
Address Nutley, NJ Phone: (201) 812-3674 Fax: (201) 812-3700
Status: Designated/Approved Designation 06-Jun-89 Marketing Approval 19-Oct-95
Generic Name: Interferon alfa-2a (recombinant)
| Trade Name: Roferon-A
|
Orphan Indication: Treatment of AIDS related Kaposi's sarcoma.
Sponsor Hoffmann-La Roche, Inc. Contact Ms. Loni da Silva
Address Nutley, NJ Phone: (201) 812-3674 Fax: (201) 812-3700
Status: Designated/Approved Designation 14-Dec-8 Marketing Approval 21-Nov-88
Generic Name: Interferon alfa-2b (recombinant)
| Trade Name: Intron A
|
Orphan Indication: Treatment of AIDS-related Kaposi's sarcoma.
Sponsor Schering Corporation Contact Dr. Alexander Giaquinto
Address Kenilworth, NJ Phone: (201) 298-4000 Fax:
Status: Designated/Approved Designation 24-Jun-87 Marketing Approval 21-Nov-88
Generic Name: Interferon beta-1a
| Trade Name: Avonex
|
Orphan Indication: Treatment of multiple sclerosis.
Sponsor Biogen, Inc. Contact Dr. Burt Adelman
Address Cambridge, MA Phone: (617) 679-2000 Fax: (617) 679-2617
Status: Designated/Approved Designation 16-Dec-9 Marketing Approval 17-May-96
Generic Name: Interferon beta-1b
| Trade Name: Betaseron
|
Orphan Indication: Treatment of multiple sclerosis.
Sponsor Chiron Corp. & Berlex Laboratories Contact Mr. Dana Redhair
Address Emeryville, CA Phone: (510) 923-2786 Fax: (510) 923-3344
Status: Designated/Approved Designation 17-Nov-8 Marketing Approval 23-Jul-93
Generic Name: Interferon gamma 1-b
| Trade Name: Actimmune
|
Orphan Indication: Treatment of chronic granulomatous disease.
Sponsor Genentech, Inc. Contact Dr. M. David MacFarlane
Address South San Francisco, CA Phone: (415) 225-1000 Fax: (415) 225-6000
Status: Designated/Approved Designation 30-Sep-8 Marketing Approval 20-Dec-90
Generic Name: Interferon gamma-1b
| Trade Name: Actimmune
|
Orphan Indication: Delaying time to disease progression in patients with severe, malignant
osteopetrosis.
Sponsor InterMune , Inc. Contact Ms. Staci Ellis
Address Brisbane, CA Phone: 4154662211 Fax: 4155080453
Status: Designated/Approved Designation 30-Sep-9 Marketing Approval 10-Feb-00
Generic Name: Iobenguane Sulfate I 131
Trade Name:
|
Orphan Indication: For use as a diagnostic adjunct in patients with pheochromocytoma.
Sponsor University of Michigan Contact Mr. David Hubers
Address Ann Arbor, MI Phone: 7349365304 Fax: 7349368182
Status: Designated/Approved/Wi Designation 14-Nov-8 Marketing Approval 21-Mar-90
thdrawn
Generic Name: Lamotrigine
| Trade Name: Lamictal
|
Orphan Indication: Treatment of Lennox-Gastaut syndrome.
Sponsor Glaxo Wellcome Research and Development Contact Dr. Elizabeth McConnell
Address Research Triangle Park, NC Phone: (919) 483-6466 Fax: (919) 483-5118
Status: Designated/Approved Designation 23-Aug-9 Marketing Approval 24-Aug-98
Generic Name: Lepirudin
| Trade Name: Refluden
|
Orphan Indication: Treatment of heparin-associated thrombocytopenia type II.
Sponsor Hoechst Marion Roussel Contact Mr. B. Randall Vestal
Address Germany, DE Phone: (919) 462-2342 Fax: (919) 380-1840
Status: Designated/Approved Designation 13-Feb-9 Marketing Approval 06-Mar-98
Generic Name: Leucovorin
| Trade Name: Leucovorin calcium
|
Orphan Indication: For rescue use after high dose methotrexate therapy in the treatment of
osteosarcoma.
Sponsor Immunex Corporation Contact Ms. Sally Gould
Address Seattle, WA Phone: (206) 389-4092 Fax:
Status: Designated/Approved Designation 17-Aug-8 Marketing Approval 31-Aug-88
Generic Name: Leucovorin
| Trade Name: Leucovorin calcium
|
Orphan Indication: For use in combination with 5-fluorouracil for the treatment of metastatic
colorectal cancer.
Sponsor Immunex Corporation Contact Ms. Sally Gould
Address Seattle, WA Phone: (206) 389-4092 Fax:
Status: Designated/Approved Designation 08-Dec-8 Marketing Approval 12-Dec-91
Generic Name: Leuprolide acetate
| Trade Name: Lupron Injection
|
Orphan Indication: Treatment of central precocious puberty.
Sponsor Tap Pharmaceuticals, Inc. Contact Dr. S. Albert Edwards
Address Deerfield, IL Phone: (708) 317-5780 Fax:
Status: Designated/Approved Designation 25-Jul-88 Marketing Approval 16-Apr-93
Generic Name: Levocarnitine
| Trade Name: Carnitor
|
Orphan Indication: Treatment of genetic carnitine deficiency.
Sponsor Sigma-Tau Pharmaceuticals, Inc. Contact Dr. Edward Helton
Address Gaithersburg, MD Phone: (301) 948-1041 Fax: (301) 948-3194
Status: Designated/Approved Designation 28-Feb-8 Marketing Approval 10-Apr-86
Generic Name: Levocarnitine
| Trade Name: Carnitor
|
Orphan Indication: Treatment of manifestations of carnitine deficiency in patients with end
stage renal disease who require dialysis.
Sponsor Sigma-Tau Pharmaceuticals, Inc. Contact Mr. A Hanzas
Address Gaithersburg, MD Phone: (301) 670-2192 Fax: (301) 948-3194
Status: Designated/Approved Designation 06-Sep-8 Marketing Approval 15-Dec-99
Generic Name: Levocarnitine
| Trade Name: Carnitor
|
Orphan Indication: Treatment of primary and secondary carnitine deficiency of genetic origin.
Sponsor Sigma-Tau Pharmaceuticals, Inc. Contact Dr. Edward Helton
Address Gaithersburg, MD Phone: (301) 948-1041 Fax: (301) 948-3194
Status: Designated/Approved Designation 26-Jul-84 Marketing Approval 16-Dec-92
Generic Name: Levomethadyl acetate hydrochloride
| Trade Name: Orlaam
|
Orphan Indication: Treatment of heroin addicts suitable for maintenance on opiate agonists.
Sponsor Biodevelopment Corporation Contact Mr. John Thomas
Address McLean, VA Phone: (703) 506-1904 Fax:
Status: Designated/Approved Designation 24-Jan-85 Marketing Approval 09-Jul-93
Generic Name: Lidocaine patch 5%
| Trade Name: Lidoderm Patch
|
Orphan Indication: For relief of allodynia (painful hypersensitivity), and chronic pain in
post-herpetic neuralgia.
Sponsor Teikoku Pharma USA, Inc. Contact Ms. Gale Sheirbon
Address Campbell, CA Phone: (408) 341-0130 Fax: (408) 615-4147
Status: Designated/Approved Designation 24-Oct-95 Marketing Approval 19-Mar-99
Generic Name: Liothyronine sodium injection
| Trade Name: Triostat
|
Orphan Indication: Treatment of myxedema coma/precoma.
Sponsor SmithKline Beecham Pharmaceuticals Contact Dr. Paula Goldberg
Address Philadelphia, PA Phone: (215) 751-4000 Fax: (215) 751-3400
Status: Designated/Approved Designation 30-Jul-90 Marketing Approval 31-Dec-91
Generic Name: Liposomal amphotericin B
| Trade Name: AmBisome
|
Orphan Indication: Treatment of visceral leishmaniasis.
Sponsor Fujisawa USA, Inc. Contact Dr. Jerry Johnson
Address Deerfield, IL Phone: (847) 317-8800 Fax: (847) 317-7286
Status: Designated/Approved Designation 06-Dec-9 Marketing Approval 11-Aug-97
Generic Name: Liposomal amphotericin B
| Trade Name: AmBisome
|
Orphan Indication: Treatment of cryptococcal meningitis.
Sponsor Fujisawa USA, Inc. Contact Dr. Jerry Johnson
Address Deerfield, IL Phone: (847) 317-8800 Fax: (847) 317-7286
Status: Designated/Approved Designation 10-Dec-9 Marketing Approval 11-Aug-97
Generic Name: Lodoxamide tromethamine
| Trade Name: Alomide Ophthalmic Solution
|
Orphan Indication: Treatment of vernal keratoconjunctivitis.
Sponsor Alcon Laboratories, Inc. Contact Dr. Robert Roehrs
Address Fort Worth, TX Phone: (817) 551-8512 Fax:
Status: Designated/Approved Designation 16-Oct-91 Marketing Approval 23-Sep-93
Generic Name: Mafenide acetate solution
| Trade Name: Sulfamylon solution
|
Orphan Indication: For use as an adjunctive topical antimicrobial agent to control bacterial
infection when used under moist dressings over meshed autografts on
excised burn wounds.
Sponsor Mylan Laboratories, Inc. Contact Mr. Frank Sisto
Address Morgantown, WV Phone: (304) 599-2595 Fax:
Status: Designated/Approved Designation 18-Jul-90 Marketing Approval 05-Jun-98
Generic Name: Mefloquine HCl
| Trade Name: Lariam
|
Orphan Indication: Prophylaxis of Plasmodium falciparum malaria which is resistant to other
available drugs.
Sponsor Hoffmann-La Roche, Inc. Contact Ms. Jeannie-Marie Skinner
Address Nutley, NJ Phone: (201) 235-5405 Fax:
Status: Designated/Approved Designation 13-Apr-88 Marketing Approval 02-May-89
Generic Name: Mefloquine HCl
| Trade Name: Lariam
|
Orphan Indication: Treatment of acute malaria due to Plasmodium falciparum and Plasmodium
vivax.
Sponsor Hoffmann-La Roche, Inc. Contact Ms. Jeannie-Marie Skinner
Address Nutley, NJ Phone: (201) 235-5405 Fax:
Status: Designated/Approved Designation 13-Apr-88 Marketing Approval 02-May-89
Generic Name: Megestrol acetate
| Trade Name: Megace
|
Orphan Indication: Treatment of patients with anorexia, cachexia, or significant weight loss
(=/>10% of baseline body weight) and confirmed diagnosis of AIDS.
Sponsor Bristol-Myers Squibb Pharmaceutical Research Contact Ms. Marygayle Ritzert
Institute
Address Evansville, IN Phone: (812) 429-5584 Fax:
Status: Designated/Approved Designation 13-Apr-88 Marketing Approval 10-Sep-93
Generic Name: Mesna
| Trade Name: Mesnex
|
Orphan Indication: For use as a prophylactic agent in reducing the incidence of
ifosfamide-induced hemorrhagic cystitis.
Sponsor Degussa Corporation Contact Mr. Ralph Venhaus
Address Ridgefield Park, NJ Phone: (201) 641-6100 Fax:
Status: Designated/Approved Designation 14-Nov-8 Marketing Approval 30-Dec-88
Generic Name: Methotrexate sodium
| Trade Name: Methotrexate
|
Orphan Indication: Treatment of osteogenic sarcoma.
Sponsor Lederle Laboratories Contact Dr. Vern De Vries
Address Pearl River, NY Phone: (914) 732-5000 Fax:
Status: Designated/Approved Designation 21-Oct-85 Marketing Approval 07-Apr-88
Generic Name: Metronidazole (topical)
| Trade Name: Metrogel
|
Orphan Indication: Treatment of acne rosacea.
Sponsor Galderma Laboratories, Inc. Contact Ms. Christine Shank
Address Fort Worth, TX Phone: (817) 263-2600 Fax: (817) 263-2667
Status: Designated/Approved Designation 22-Oct-87 Marketing Approval 22-Nov-88
Generic Name: Midodrine HCl
| Trade Name: Amatine
|
Orphan Indication: Treatment of patients with symptomatic orthostatic hypotension.
Sponsor Schier Ridgewood F.K.A. (Roberts Contact Mr. Drew Karlan
Pharmaceutical Corp.)
Address Eatontown, NJ Phone: 8008282088 Fax:
Status: Designated/Approved Designation 05-Dec-9 Marketing Approval 06-Sep-96
Generic Name: Mitoxantrone
| Trade Name: Novantrone
|
Orphan Indication: Treatment of secondary-progressive multiple sclerosis.
Sponsor Immunex Corporation Contact Mr. Mark Gauthier
Address Seattle, WA Phone: 2063816266 Fax:
Status: Designated/Approved Designation 13-Aug-9 Marketing Approval 13-Oct-00
Generic Name: Mitoxantrone
| Trade Name: Novantrone
|
Orphan Indication: Treatment of progressive-relapsing multiple sclerosis.
Sponsor Immunex Corporation Contact Mr. Mark Gauthier
Address Seattle, WA Phone: (206) 389-4066 Fax:
Status: Designated/Approved Designation 13-Aug-9 Marketing Approval 13-Oct-00
Generic Name: Mitoxantrone
| Trade Name: Novantrone
|
Orphan Indication: Treatment of hormone refractory prostate cancer.
Sponsor Immunex Corporation Contact Mr. Mark Gauthier
Address Seattle, WA Phone: (206) 389-4066 Fax: (206) 587-0606
Status: Designated/Approved Designation 21-Aug-9 Marketing Approval 13-Nov-96
Generic Name: Mitoxantrone HCl
| Trade Name: Novantrone
|
Orphan Indication: Treatment of acute myelogenous leukemia, also referred to as acute
nonlymphocytic leukemia.
Sponsor Lederle Laboratories Contact Dr. Vern DeVries
Address Pearl River, NY Phone: (914) 732-5000 Fax:
Status: Designated/Approved Designation 13-Jul-87 Marketing Approval 23-Dec-87
Generic Name: Modafinil
| Trade Name: Provigil
|
Orphan Indication: Treatment of excessive daytime sleepiness in narcolepsy.
Sponsor Cephalon, Inc. Contact Mr. Paul Kirsch
Address West Chester, PA Phone: (610) 738-6122 Fax: (610) 344-0065
Status: Designated/Approved Designation 15-Mar-93 Marketing Approval 24-Dec-98
Generic Name: Monooctanoin
| Trade Name: Moctanin
|
Orphan Indication: For dissolution of cholesterol gallstones retained in the common bile duct.
Sponsor Ethitek Pharmaceuticals, Inc. Contact Dr. Alan Raff
Address Skokie, IL Phone: (708) 966-2200 Fax:
Status: Designated/Approved Designation 30-May-8 Marketing Approval 31-Oct-85
4
Generic Name: Morphine sulfate concentrate (preservative free)
| Trade Name: Infumorph
|
Orphan Indication: For use in microinfusion devices for intraspinal administration in the
treatment of intractable chronic pain.
Sponsor Elkins-Sinn, Inc. Contact Ms. Thelma Hilibrand
Address Cherry Hill, NJ Phone: (609) 424-3700 Fax:
Status: Designated/Approved Designation 12-Jul-90 Marketing Approval 19-Jul-91
Generic Name: Nafarelin acetate
| Trade Name: Synarel Nasal Solution
|
Orphan Indication: Treatment of central precocious puberty.
Sponsor Syntex (USA), Inc. Contact Dr. Michael Perry
Address Palo Alto, CA Phone: (415) 354-7453 Fax:
Status: Designated/Approved Designation 20-Jul-88 Marketing Approval
Generic Name: Naltrexone HCl
| Trade Name: Trexan
|
Orphan Indication: For blockade of the pharmacological effects of exogenously administered
opioids as an adjunct to the maintenance of the opioid-free state in
detoxified formerly opioid-dependent individuals.
Sponsor DuPont Pharmaceuticals Contact Mr. Edward Adams
Address Wilmington, DE Phone: (302) 992-5094 Fax:
Status: Designated/Approved Designation 11-Mar-85 Marketing Approval 30-Nov-84
Generic Name: Nitisinone
| Trade Name: Orfadin
|
Orphan Indication: Treatment of tyrosinemia type 1.
Sponsor Swedish Orphan AB Contact Dr. Ronald Leonardi
Address Sweden, SE Phone: (858) 586-0751 Fax: (858) 586-1108
Status: Designated/Approved Designation 16-May-9 Marketing Approval 18-Jan-02
5
Generic Name: Nitric oxide
| Trade Name: INOmax
|
Orphan Indication: Treatment of persistent pulmonary hypertension in the newborn.
Sponsor INO Therapeutics, Inc. Contact Mr. Richard Williams
Address Clinton, NJ Phone: (908) 238-6622 Fax: (908) 238-0779
Status: Designated/Approved Designation 22-Jun-93 Marketing Approval 23-Dec-99
Generic Name: Octreotide
| Trade Name: Sandostatin LAR
|
Orphan Indication: Treatment of severe diarrhea and flushing associated with malignant
carcinoid tumors.
Sponsor Novartis Pharmaceuticals Corporation Contact Ms. Eileen Ryan
Address East Hanover, NJ Phone: (973) 781-7661 Fax: (973) 781-6325
Status: Designated/Approved Designation 24-Aug-9 Marketing Approval 25-Nov-98
Generic Name: Octreotide
| Trade Name: Sandostatin LAR
|
Orphan Indication: Treatment of acromegaly.
Sponsor Novartis Pharmaceuticals Corporation Contact Ms. Eileen Ryan
Address East Hanover, NJ Phone: (973) 781-7661 Fax: (973) 781-6325
Status: Designated/Approved Designation 24-Aug-9 Marketing Approval 25-Nov-98
Generic Name: Octreotide
| Trade Name: Sandostatin LAR
|
Orphan Indication: Treatment of diarrhea associated with vasoactive intestinal peptide tumors
(VIPoma).
Sponsor Novartis Pharmaceuticals Corporation Contact Ms. Eileen Ryan
Address East Hanover, NJ Phone: (973) 781-7661 Fax: (973) 781-6325
Status: Designated/Approved Designation 24-Aug-9 Marketing Approval 25-Nov-98
Generic Name: Ofloxacin
| Trade Name: Ocuflox Ophthalmic Solution
|
Orphan Indication: Treatment of bacterial corneal ulcers.
Sponsor Allergan, Inc. Contact Ms. Elizabeth Bancroft
Address Irvine, CA Phone: (714) 752-4500 Fax:
Status: Designated/Approved Designation 18-Apr-91 Marketing Approval 22-May-96
Generic Name: Oprelvekin
| Trade Name: Neumega
|
Orphan Indication: Prevention of severe chemotherapy-induced thrombocytopenia.
Sponsor Genetics Institute, Inc. Contact Ms. Ingride Bartinique
Address Cambridge, MA Phone: (617) 498-8652 Fax: (617) 498-8876
Status: Designated/Approved Designation 17-Dec-9 Marketing Approval 25-Nov-97
Generic Name: Paclitaxel
| Trade Name: Taxol
|
Orphan Indication: Treatment of AIDS-related Kaposi's sarcoma.
Sponsor Bristol-Myers Squibb Pharmaceutical Research Contact Ms. Cheryl Anderson
Institute
Address Wallingford, CT Phone: (203) 284-6083 Fax: (203) 284-7630
Status: Designated/Approved Designation 25-Mar-97 Marketing Approval 04-Aug-97
Generic Name: Pegademase bovine
| Trade Name: Adagen
|
Orphan Indication: For enzyme replacement therapy for ADA deficiency in patients with
severe combined immunodeficiency.
Sponsor Enzon, Inc. Contact Mr. Brian Bollwage
Address Piscataway, NJ Phone: (908) 980-4500 Fax: (908) 980-5911
Status: Designated/Approved Designation 29-May-8 Marketing Approval 21-Mar-90
4
Generic Name: Pegaspargase
| Trade Name: Oncaspar
|
Orphan Indication: Treatment of acute lymphocytic leukemia.
Sponsor Enzon, Inc. Contact Mr. Brian Bollwage
Address Piscataway, NJ Phone: (908) 980-4500 Fax: (908) 980-5911
Status: Designated/Approved Designation 20-Oct-89 Marketing Approval 01-Feb-94
Generic Name: Pentamidine isethionate
| Trade Name: Pentam 300
|
Orphan Indication: Treatment of Pneumocystis carinii pneumonia.
Sponsor Fujisawa USA, Inc. Contact Dr. Jerry Johnson
Address Deerfield, IL Phone: (847) 317-8800 Fax: (847) 317-7286
Status: Designated/Approved Designation 28-Feb-8 Marketing Approval 16-Oct-84
Generic Name: Pentamidine isethionate
| Trade Name: Nebupent
|
Orphan Indication: Prevention of Pneumocystis carinii pneumonia in patients at high risk of
developing this disease.
Sponsor Fujisawa USA, Inc. Contact Dr. Jerry Johnson
Address Deerfield, IL Phone: (847) 317-8800 Fax: (847) 317-7286
Status: Designated/Approved Designation 12-Jan-88 Marketing Approval 15-Jun-89
Generic Name: Pentastarch
| Trade Name: Pentaspan
|
Orphan Indication: As an adjunct in leukapheresis to improve the harvesting and increase the
yield of leukocytes by centrifugal means.
Sponsor Du Pont Pharmaceuticals Contact Mr. Edward Adams
Address Wilmington, DE Phone: (302) 992-5094 Fax:
Status: Designated/Approved Designation 28-Aug-8 Marketing Approval 19-May-87
Generic Name: Pentosan polysulfate sodium
| Trade Name: Elmiron
|
Orphan Indication: Treatment of interstitial cystitis.
Sponsor Alza Corporation Contact Ms. Mirka Dunn
Address Palo Alto, CA Phone: (650) 564-2524 Fax: (650) 564-2581
Status: Designated/Approved Designation 07-Aug-8 Marketing Approval 26-Sep-96
Generic Name: Pentostatin for injection
| Trade Name: Nipent
|
Orphan Indication: Treatment of hairy cell leukemia.
Sponsor SuperGen, Inc. Contact Dr. Sam Boddapati
Address Dublin, CA Phone: 9255600100 Fax: 9255516472
Status: Designated/Approved Designation 10-Sep-8 Marketing Approval 11-Oct-91
Generic Name: Pilocarpine
| Trade Name: Salagen
|
Orphan Indication: Treatment of xerostomia induced by radiation therapy for head and neck
cancer.
Sponsor MGI Pharma, Inc. Contact Dr. Jo Gustafson
Address Bloomington, MN Phone: (612) 346-4722 Fax: (612) 346-4800
Status: Designated/Approved Designation 24-Sep-9 Marketing Approval 22-Mar-94
Generic Name: Pilocarpine HCl
| Trade Name: Salagen
|
Orphan Indication: Treatment of xerostomia and keratoconjunctivitis sicca in Sjogren's
syndrome patients.
Sponsor MGI Pharma, Inc. Contact Dr. Jo Gustafson
Address Bloomington, MN Phone: (612) 346-4722 Fax: (612) 346-4800
Status: Designated/Approved Designation 28-Feb-9 Marketing Approval 11-Feb-98
Generic Name: Polifeprosan 20 with carmustine
| Trade Name: Gliadel
|
Orphan Indication: Treatment of malignant glioma.
Sponsor Guilford Pharmaceuticals, Inc. Contact Mr. Ross Laderman
Address Baltimore, MD Phone: (410) 631-6306 Fax: (410) 631-6338
Status: Designated/Approved Designation 13-Dec-8 Marketing Approval 23-Sep-96
Generic Name: Porfimer sodium
| Trade Name: Photofrin
|
Orphan Indication: For the photodynamic therapy of patients with primary or recurrent
obstructing (either partially or completely) esophageal carcinoma.
Sponsor QLT Phototherapeutics, Inc. Contact Mr. Louis Gura
Address Pearl River, NY Phone: (914) 732-5000 Fax:
Status: Designated/Approved Designation 06-Jun-89 Marketing Approval 27-Dec-95
Generic Name: Potassium citrate
| Trade Name: Urocit-K
|
Orphan Indication: Prevention of calcium renal stones in patients with hypocitraturia.
Sponsor University of Texas Health Science Center at Contact Dr. Charles Y.C. Pak
Address Dallas, TX Phone: (214) 688-2100 Fax:
Status: Designated/Approved Designation 16-Sep-8 Marketing Approval 30-Aug-85
Generic Name: Potassium citrate
| Trade Name: Urocit-K
|
Orphan Indication: For avoidance of the complication of calcium stone formation in patients
with uric lithiasis.
Sponsor University of Texas Health Science Center at Contact Dr. Charles Y.C. Pak
Address Dallas, TX Phone: (214) 688-2100 Fax:
Status: Designated/Approved Designation 12-Sep-8 Marketing Approval 30-Aug-85
Generic Name: Potassium citrate
| Trade Name: Urocit-K
|
Orphan Indication: Prevention of uric acid nephrolithiasis.
Sponsor University of Texas Health Science Center at Contact Dr. Charles Y.C. Pak
Address Dallas, TX Phone: (214) 688-2100 Fax:
Status: Designated/Approved Designation 01-Nov-8 Marketing Approval 30-Aug-85
Generic Name: Pulmonary surfactant replacement, porcine
| Trade Name: Curosurf
|
Orphan Indication: For the treatment and prevention of respiratory distress syndrome in
premature infants.
Sponsor Dey Laboratories Contact Ms. Kim Carneal
Address Napa, CA Phone: (707) 224-3200 Fax: 7072241364
Status: Designated/Approved Designation 02-Aug-9 Marketing Approval
Generic Name: Respiratory syncytial virus immune globulin (Human)
| Trade Name: Respigam
|
Orphan Indication: Prophylaxis of respiratory syncytial virus lower respiratory tract infections
in infants and young children at high risk of RSV disease.
Sponsor MedImmune & Massachussetts Public Health Contact Mr. Bogdan Dziurzynski
Biologics Labs.
Address Gaithersburg, MD Phone: (301) 417-0770 Fax: (301) 417-0770
Status: Designated/Approved Designation 27-Sep-9 Marketing Approval 18-Jan-96
Generic Name: Rho (D) immune globulin intravenous (human)
| Trade Name: WinRho SD
|
Orphan Indication: Treatment of immune thrombocytopenic purpura.
Sponsor Rh Pharmaceuticals, Inc. Contact Mr. Kelvin Li
Address Canada R3T 2N2, CA Phone: (301) 770-3099 Fax:
Status: Designated/Approved Designation 09-Nov-9 Marketing Approval 24-Mar-95
Generic Name: Rifabutin
| Trade Name: Mycobutin
|
Orphan Indication: Prevention of disseminated Mycobacterium avium complex disease in
patients with advanced HIV infection.
Sponsor Adria Laboratories, Inc. Contact Mr. Douglas Jones
Address Columbus, OH Phone: (614) 764-8100 Fax: (614) 764-8102
Status: Designated/Approved Designation 18-Dec-8 Marketing Approval 23-Dec-92
Generic Name: Rifampin
| Trade Name: Rifadin I.V.
|
Orphan Indication: For antituberculosis treatment where use of the oral form of the drug is not
feasible.
Sponsor Hoechst Marion Roussel Contact Dr. Jack Dunn
Address Kansas City, MO Phone: (816) 966-5000 Fax:
Status: Designated/Approved Designation 09-Dec-8 Marketing Approval 25-May-89
Generic Name: Rifampin, isoniazid, pyrazinamide
| Trade Name: Rifater
|
Orphan Indication: For the short-course treatment of tuberculosis.
Sponsor Hoechst Marion Roussel Contact Dr. Jack Dunn
Address Kansas City, MO Phone: (816) 966-5000 Fax:
Status: Designated/Approved Designation 12-Sep-8 Marketing Approval 31-May-94
Generic Name: Rifapentine
| Trade Name: Priftin
|
Orphan Indication: Treatment of pulmonary tuberculosis.
Sponsor Hoechst Marion Roussel Contact Ms. Libby Hayes
Address Kansas City, MO Phone: (816) 966-7185 Fax:
Status: Designated/Approved Designation 09-Jun-95 Marketing Approval 22-Jun-98
Generic Name: Riluzole
| Trade Name: Rilutek
|
Orphan Indication: Treatment of amyotrophic lateral sclerosis.
Sponsor Rhone-Poulenc Rorer Pharmaceuticals, Inc. Contact Dr. Thomas Donnelly, Jr.
Address Collegeville, PA Phone: (610) 454-3023 Fax: (610) 454-5299
Status: Designated/Approved Designation 16-Mar-93 Marketing Approval 12-Dec-95
Generic Name: Rituximab
| Trade Name: Rituxan
|
Orphan Indication: Treatment of non-Hodgkin's B-cell lymphoma.
Sponsor IDEC Pharmaceuticals Corporation Contact Ms. Alice Wei
Address San Diego, CA Phone: (858) 431-8920 Fax: (858) 431-8889
Status: Designated/Approved Designation 13-Jun-94 Marketing Approval 26-Nov-97
Generic Name: Sacrosidase
| Trade Name: Sucraid
|
Orphan Indication: Treatment of congenital sucrase-isomaltase deficiency.
Sponsor Orphan Medical, Inc. Contact Dr. Dayton Reardon
Address Minnetonka, MN Phone: (612) 513-6900 Fax: (612) 541-9209
Status: Designated/Approved Designation 10-Dec-9 Marketing Approval 09-Apr-98
Generic Name: Sargramostim
| Trade Name: Leukine
|
Orphan Indication: Treatment of neutropenia associated with bone marrow transplant, for the
treatment of graft failure and delay of engraftment, and for the promotion
of early engraftment.
Sponsor Immunex Corporation Contact Ms. Sally Gould
Address Seattle, WA Phone: (206) 587-0430 Fax:
Status: Designated/Approved Designation 03-May-9 Marketing Approval 05-Mar-91
0
Generic Name: Sargramostim
| Trade Name: Leukine
|
Orphan Indication: To reduce neutropenia and leukopenia and decrease the incidence of death
due to infection in patients with acute myelogenous leukemia.
Sponsor Immunex Corporation Contact Ms. Sally Gould
Address Seattle, WA Phone: (206) 587-0430 Fax:
Status: Designated/Approved Designation 06-Mar-95 Marketing Approval 15-Sep-95
Generic Name: Satumomab pendetide
| Trade Name: Oncoscint CR/OV
|
Orphan Indication: Detection of ovarian carcinoma.
Sponsor Cytogen Corporation Contact Dr. Melvin Fisher
Address Princeton, NJ Phone: (609) 987-8200 Fax:
Status: Designated/Approved Designation 25-Sep-8 Marketing Approval 29-Dec-92
Generic Name: Selegiline HCl
| Trade Name: Eldepryl
|
Orphan Indication: As an adjuvant to levodopa and carbidopa treatment of idiopathic
Parkinson's disease (paralysis agitans), postencephalitic Parkinsonism, and
symptomatic Parkinsonism.
Sponsor Somerset Pharmaceuticals, Inc. Contact Mr. John Saenger
Address Tampa, FL Phone: (813) 223-7677 Fax:
Status: Designated/Approved Designation 07-Nov-8 Marketing Approval 05-Jun-89
Generic Name: Sermorelin acetate
| Trade Name: Geref
|
Orphan Indication: Treatment of idiopathic or organic growth hormone deficiency in children
with growth failure.
Sponsor Serono Laboratories, Inc. Contact Mr. Thomas Lang
Address Norwell, MA Phone: (617) 982-9000 Fax: (617) 871-6754
Status: Designated/Approved Designation 14-Sep-8 Marketing Approval 26-Sep-97
Generic Name: sodium phenylbutyrate
| Trade Name: Buphenyl
|
Orphan Indication: Treatment of urea cycle disorders: carbamylphosphate synthetase
deficiency, ornithine transcarbamylase deficiency, and arginiosuccinic acid
synthetase deficiency.
Sponsor Medicis Pharmaceutical Corp. Contact Ms. Lynn Hansen
Address Scottsdale, AZ Phone: 6028083813 Fax: 6028080822
Status: Designated/Approved Designation 22-Nov-9 Marketing Approval 30-Apr-96
Generic Name: Somatrem for injection
| Trade Name: Protropin
|
Orphan Indication: For long-term treatment of children who have growth failure due to a lack of
adequate endogenous growth hormone secretion.
Sponsor Genentech, Inc. Contact Dr. M. David MacFarlane
Address South San Francisco, CA Phone: (415) 225-1000 Fax: (415) 225-6000
Status: Designated/Approved Designation 09-Dec-8 Marketing Approval 17-Oct-85
Generic Name: Somatropin
| Trade Name: Nutropin
|
Orphan Indication: For use in the long-term treatment of children who have growth failure due
to a lack of adequate endogenous growth hormone secretion.
Sponsor Genentech, Inc. Contact Dr. M. David MacFarlane
Address South San Francisco, CA Phone: (415) 225-1000 Fax: (415) 225-6000
Status: Designated/Approved Designation 06-Mar-87 Marketing Approval 17-Oct-85
Generic Name: Somatropin
| Trade Name: Genotropin
|
Orphan Indication: Treatment of adults with growth hormone deficiency.
Sponsor Pharmacia & Upjohn Contact Mr. Gregory Shawaryn
Address Kalamazoo, MI Phone: (616) 833-8239 Fax: (616) 833-8237
Status: Designated/Approved Designation 06-Sep-9 Marketing Approval 31-Oct-97
Generic Name: Somatropin
| Trade Name: Humatrope
|
Orphan Indication: Treatment of short stature associated with Turner syndrome.
Sponsor Eli Lilly and Company Contact Dr. Timothy Franson
Address Indianapolis, IN Phone: (317) 276-2000 Fax:
Status: Designated/Approved Designation 08-May-9 Marketing Approval 30-Dec-96
0
Generic Name: Somatropin (rDNA origin)
| Trade Name: Nutropin Depot
|
Orphan Indication: Long-term treatment of children who have growth failure due to a lack of
adequate endogenous growth hormone secretion.
Sponsor Genentech, Inc. Contact Dr. Robert Garnick
Address South San Francisco, CA Phone: (650) 225-1202 Fax: (650) 225-5927
Status: Designated/Approved Designation 28-Oct-99 Marketing Approval
Generic Name: Somatropin (rDNA origin)
| Trade Name: Saizen
|
Orphan Indication: Treatment of idiopathic or organic growth hormone deficiency in children
with growth failure.
Sponsor Serono Laboratories, Inc. Contact Ms. Pamela Williamson
Joyce
Address Norwell, MA Phone: (800) 283-8088 Fax: (617) 871-6754
Status: Designated/Approved Designation 06-Mar-87 Marketing Approval
Generic Name: Somatropin (rDNA origin) injection
| Trade Name: Norditropin
|
Orphan Indication: Treatment of growth failure in children due to inadequate growth hormone
secretion.
Sponsor Novo Nordisk Pharmaceuticals Contact Mr. Lawrence Rosania
Address Princeton, NJ Phone: (609) 987-5847 Fax: (609) 921-8082
Status: Designated/Approved Designation 10-Jul-87 Marketing Approval
Generic Name: Somatropin [rDNA]
| Trade Name: Genotropin
|
Orphan Indication: Treatment of short stature in patients with Prader-Willi syndrome.
Sponsor Pharmacia & Upjohn Contact Ms. Cynthia Blanchard
Address Kalamazoo, MI Phone: (616) 833-6717 Fax:
Status: Designated/Approved Designation 06-Jul-99 Marketing Approval 20-Jun-00
Generic Name: somatropin [rDNA]
| Trade Name: Genotropin
|
Orphan Indication: Treatment of growth failure in children who were born small for gestational
age.
Sponsor Pharmacia and Upjohn Company Contact Mr. Daniel Chirby
Address Kalamazoo, MI Phone: (616) 833-9411 Fax:
Status: Designated/Approved Designation 27-Dec-0 Marketing Approval 25-Jul-01
Generic Name: Somatropin for injection
| Trade Name: Serostim
|
Orphan Indication: Treatment of AIDS-associated catabolism/weight loss.
Sponsor Serono Laboratories, Inc. Contact Mr. Thomas Lang
Address Norwell, MA Phone: (617) 982-9000 Fax: (617) 871-6754
Status: Designated/Approved Designation 15-Nov-9 Marketing Approval 23-Aug-96
Generic Name: Somatropin for injection
| Trade Name: Humatrope
|
Orphan Indication: For the long-term treatment of children who have growth failure due to
inadequate secretion of normal endogenous growth hormone.
Sponsor Eli Lilly and Company Contact Dr. Timothy Franson
Address Indianapolis, IN Phone: (317) 276-2000 Fax:
Status: Designated/Approved Designation 12-Jun-86 Marketing Approval 08-Mar-87
Generic Name: Somatropin for injection
| Trade Name: Nutropin
|
Orphan Indication: Treatment of short stature associated with Turner's syndrome.
Sponsor Genentech, Inc. Contact Dr. M. David MacFarlane
Address South San Francisco, CA Phone: (415) 225-1000 Fax: (415) 225-6000
Status: Designated/Approved Designation 23-Mar-89 Marketing Approval 30-Dec-96
Generic Name: Somatropin for injection
| Trade Name: Nutropin
|
Orphan Indication: As replacement therapy for growth hormone deficiency in adults after
epiphyseal closure.
Sponsor Genentech, Inc. Contact Dr. M. David MacFarlane
Address South San Francisco, CA Phone: (415) 225-1000 Fax: (415) 225-6000
Status: Designated/Approved Designation 18-Nov-9 Marketing Approval 15-Dec-97
Generic Name: Somatropin for injection
| Trade Name: Nutropin
|
Orphan Indication: Treatment of growth retardation associated with chronic renal failure.
Sponsor Genentech, Inc. Contact Dr. M. David MacFarlane
Address South San Francisco, CA Phone: (415) 225-1000 Fax: (415) 225-6000
Status: Designated/Approved Designation 04-Aug-8 Marketing Approval 17-Nov-93
Generic Name: Sotalol HCl
| Trade Name: Betapace
|
Orphan Indication: Treatment of life-threatening ventricular tachyarrhythmias.
Sponsor Berlex Laboratories, Inc. Contact Dr. Suzanne Hampton
Address Wayne, NJ Phone: (201) 305-5288 Fax:
Status: Designated/Approved Designation 23-Sep-8 Marketing Approval 30-Oct-92
Generic Name: Sterile talc powder
| Trade Name: Sclerosol Intrapleural Aerosol
|
Orphan Indication: Treatment of malignant pleural effusion.
Sponsor Bryan Corporation Contact Mr. Frank Abrano
Address Woburn, MA Phone: (781) 935-0004 Fax: (781) 935-7602
Status: Designated/Approved Designation 18-Sep-9 Marketing Approval 24-Dec-97
Generic Name: Succimer
| Trade Name: Chemet capsules
|
Orphan Indication: Treatment of lead poisoning in children.
Sponsor Bock Pharmacal Company Contact Mr. David Byron
Address Saint Louis, MO Phone: (800) 727-2625 Fax:
Status: Designated/Approved Designation 09-May-8 Marketing Approval 22-Dec-88
4
Generic Name: Sulfadiazine
Trade Name:
|
Orphan Indication: For use in combination with pyrimethamine for the treatment of Toxoplasma
gondii
encephalitis in patients with and without AIDS.
Sponsor Eon Labs Manufacturing, Inc. Contact Ms. Carol Frankel
Address Laurelton, NY Phone: (212) 755-2339 Fax:
Status: Designated/Approved Designation 14-Mar-94 Marketing Approval 29-Jul-94
Generic Name: Surface active extract of saline lavage of bovine lungs
| Trade Name: Infasurf
|
Orphan Indication: Treatment and prevention of respiratory failure due to pulmonary surfactant
deficiency in preterm infants.
Sponsor ONY, Inc. Contact Dr. Edmund Egan
Address Amherst, NY Phone: (716) 636-9096 Fax:
Status: Designated/Approved Designation 07-Jun-85 Marketing Approval
Generic Name: Temozolomide
| Trade Name: Temodar
|
Orphan Indication: Treatment of recurrent malignant glioma.
Sponsor Schering-Plough Research Institute Contact Dr. Joseph Lamendola
Address Kenilworth, NJ Phone: (908) 298-2628 Fax:
Status: Designated/Approved Designation 05-Oct-98 Marketing Approval 11-Aug-99
Generic Name: Teniposide
| Trade Name: Vumon for injection
|
Orphan Indication: Treatment of refractory childhood acute lymphocytic leukemia.
Sponsor Bristol-Myers Squibb Pharmaceutical Research Contact Dr. Douglas Kriesel
Institute
Address Wallingford, CT Phone: (203) 284-6051 Fax:
Status: Designated/Approved Designation 01-Nov-8 Marketing Approval 14-Jul-92
Generic Name: Teriparatide
| Trade Name: Parathar
|
Orphan Indication: Diagnostic agent to assist in establishing the diagnosis in patients
presenting with clinical and laboratory evidence of hypocalcemia due to
either hypoparathyroidism or pseudohypoparathyroidism.
Sponsor Rhone-Poulenc Rorer Pharmaceuticals, Inc. Contact Dr. Thomas Donnelly
Address Collegeville, PA Phone: (610) 454-3023 Fax:
Status: Designated/Approved Designation 09-Jan-87 Marketing Approval 23-Dec-87
Generic Name: Thalidomide
| Trade Name: Thalomid
|
Orphan Indication: Treatment of erythema nodosum leprosum.
Sponsor Celgene Corporation Contact Dr. Steve Thomas
Address Warren, NJ Phone: (732) 805-3914 Fax: (732) 271-4184
Status: Designated/Approved Designation 26-Jul-95 Marketing Approval 16-Jul-98
Generic Name: Thyrotropin alpha
| Trade Name: Thyrogen
|
Orphan Indication: As an adjunct in the diagnosis of thyroid cancer.
Sponsor Genzyme Corporation Contact Ms. Allison Lawton
Address Cambridge, MA Phone: (617) 252-7500 Fax: (617) 252-7600
Status: Designated/Approved Designation 24-Feb-9 Marketing Approval 30-Nov-98
Generic Name: Tiopronin
| Trade Name: Thiola
|
Orphan Indication: Prevention of cystine nephrolithiasis in patients with homozygous
cystinuria.
Sponsor Pak, Charles Y.C. M.D. Contact Dr. Charles Y.C. Pak
Address Dallas, TX Phone: 6214) 688-2100 Fax:
Status: Designated/Approved Designation 17-Jan-86 Marketing Approval 11-Aug-88
Generic Name: Tobramycin for inhalation
| Trade Name: TOBI
|
Orphan Indication: Treatment of bronchopulmonary infections of Pseudomonas aeruginosa in
cystic fibrosis patients.
Sponsor Pathogenesis Corporation Contact Dr. A. Bruce Montgomery
Address Seattle, WA Phone: (206) 467-8100 Fax: (206) 282-5065
Status: Designated/Approved Designation 13-Oct-94 Marketing Approval 22-Dec-97
Generic Name: Topiramate
| Trade Name: Topamax
|
Orphan Indication: Treatment of Lennox-Gastaut syndrome.
Sponsor R. W. Johnson Pharmaceutical Research Institute Contact Ms. Catherine Glamkowski
Address Raritan, NJ Phone: (908) 704-5360 Fax:
Status: Designated/Approved Designation 25-Nov-9 Marketing Approval 28-Aug-01
Generic Name: Toremifene
| Trade Name: Fareston
|
Orphan Indication: Hormonal therapy of metastatic carcinoma of the breast.
Sponsor Orion Corporation Contact Dr. Daniel Azarnoff
Address Finland, Phone: (415) 340-8222 Fax:
Status: Designated/Approved Designation 19-Sep-9 Marketing Approval 29-May-97
Generic Name: Tranexamic acid
| Trade Name: Cyklokapron
|
Orphan Indication: Treatment of patients with congenital coagulopathies who are undergoing
surgical procedures (e.g. dental extractions).
Sponsor Pharmacia Inc. Contact Mr. Robert Watson
Address Columbus, OH Phone: (614) 764-8100 Fax:
Status: Designated/Approved/Wi Designation 29-Oct-85 Marketing Approval 30-Dec-86
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Generic Name: Tretinoin
| Trade Name: Vesanoid
|
Orphan Indication: Treatment of acute promyelocytic leukemia.
Sponsor Hoffmann-La Roche, Inc. Contact Dr. David Parkinson
Address Nutley, NJ Phone: (201) 496-8798 Fax:
Status: Designated/Approved Designation 24-Oct-90 Marketing Approval 22-Nov-95
Generic Name: Trientine HCl
| Trade Name: Syprine
|
Orphan Indication: Treatment of patients with Wilson's disease who are intolerant, or
inadequately responsive to penicillamine.
Sponsor Merck Sharp & Dohme Research Contact Dr. Robert Zeldin
Address West Point, PA Phone: (610) 397-3149 Fax:
Status: Designated/Approved Designation 24-Dec-8 Marketing Approval 08-Nov-85
Generic Name: Trimetrexate glucuronate
| Trade Name: Neutrexin
|
Orphan Indication: Treatment of Pneumocystis carinii pneumonia in AIDS patients.
Sponsor Medimmune Oncology, Inc. Contact Ms. Eve Damiano
Address West Conshohocken, PA Phone: (610) 832-4580 Fax: (610) 832-4571
Status: Designated/Approved Designation 15-May-8 Marketing Approval 17-Dec-93
6
Generic Name: Urofollitropin
| Trade Name: Metrodin
|
Orphan Indication: For induction of ovulation in patients with polycystic ovarian disease who
have an elevated LH/FSH ratio and who have failed to respond to adequate
clomiphene citrate therapy.
Sponsor Serono Laboratories, Inc. Contact Mr. Thomas Lang
Address Norwell, MA Phone: (617) 982-9000 Fax: (617) 871-6754
Status: Designated/Approved Designation 25-Nov-8 Marketing Approval 18-Sep-86
Generic Name: Ursodiol
| Trade Name: URSO
|
Orphan Indication: Treatment of patients with primary biliary cirrhosis.
Sponsor Axcan Pharma Inc. Contact Ms. Quyen Vinh
Address Canada, CA Phone: 4504675138 Fax: (514) 669-5161
Status: Designated/Approved Designation 20-Jun-91 Marketing Approval 10-Dec-97
Generic Name: Valrubicin
| Trade Name: Valstar
|
Orphan Indication: Treatment of carcinoma in situ of the urinary bladder.
Sponsor Anthra Pharmaceuticals, Inc. Contact Dr. Nabila Turjman
Address Princeton, NJ Phone: (609) 514-1060 Fax: 6095141360
Status: Designated/Approved Designation 23-May-9 Marketing Approval 25-Sep-98
4
Generic Name: Zalcitabine
| Trade Name: Hivid
|
Orphan Indication: Treatment of AIDS.
Sponsor Hoffmann-La Roche, Inc. Contact Ms. Lisa Luther
Address Nutley, NJ Phone: (201) 235-4135 Fax:
Status: Designated/Approved Designation 28-Jun-88 Marketing Approval 19-Jun-92
Generic Name: Zidovudine
| Trade Name: Retrovir
|
Orphan Indication: Treatment of AIDS.
Sponsor Glaxo Wellcome Inc. Contact Dr. Michael Dalton
Address Research Triangle Park, NC Phone: (919) 483-2100 Fax:
Status: Designated/Approved Designation 17-Jul-85 Marketing Approval 19-Mar-87
Generic Name: Zidovudine
| Trade Name: Retrovir
|
Orphan Indication: Treatment of AIDS related complex.
Sponsor Glaxo Wellcome Inc. Contact Dr. Michael Dalton
Address Research Triangle Park, NC Phone: (919) 483-2100 Fax:
Status: Designated/Approved Designation 12-May-8 Marketing Approval 19-Mar-87
7
Generic Name: Zinc acetate
| Trade Name: Galzin
|
Orphan Indication: Treatment of Wilson's disease.
Sponsor Lemmon Company Contact Ms. Deborah Jaskot
Address Kulpsville, PA Phone: (215) 256-8400 Fax: (215) 721-8796
Status: Designated/Approved Designation 06-Nov-8 Marketing Approval 28-Jan-97
Generic Name: Zoledronate
| Trade Name: Zometa, Zabel
|
Orphan Indication: Treatment of tumor induced hypercalcemia.
Sponsor Novartis Pharmaceuticals Corp. Contact Ms. Ellen Cutler
Address East Hanover, NJ Phone: (973) 781-8180 Fax: (973) 781-6325
Status: Designated/Approved Designation 18-Aug-0 Marketing Approval 20-Aug-01