FDA
TALK PAPER

Food and Drug Administration
U.S. Department of Health and Human Services
Public Health Service 5600 Fishers Lane Rockville, MD 20857
FDA Talk Papers are prepared by the Press Office to guide FDA personnel in responding with consistency and accuracy to questions from the public on subjects of current interest. Talk Papers are subject to change as more information becomes available. 


T97-38                        Lenore Gelb:      301-827-6242
August 25, 1997               
                              Consumer Hotline: 800-532-4440


               FDA GRANTS ACCELERATED APPROVAL TO 
                HELP REPAIR DAMAGED KNEE CARTILAGE

     The Food and Drug Administration today approved a new
product that uses a patient's own cartilage cells in a surgical
procedure to repair cartilage damage in the knee.  The FDA is
approving these cartilage cells, known as autologous cultured
chondrocytes, to treat injuries to the end of the thigh bone
behind the kneecap, but not for patients with damage to the
kneecap or lower leg bones, or arthritis. 
     This product, also called Carticel, is the first one
approved under FDA's new document for industry, often called the
manipulated autologous structure (MAS) guidance.  It deals with
products comprised of living human cells manipulated outside the
body and returned to the patient for structural repair or
reconstruction.  Treatment with this cell therapy includes
harvesting and isolating normal cartilage cells from the knee,
culturing and expanding them in the laboratory and then
implanting them in the knee.  
     Carticel is generally used along with other procedures,
including the cutting away of damaged or dead tissue and the 
surgical addition of tissue taken from the patient's bones to 
cover the defect. A thorough and extensive rehabilitation
program, including leg exercises, is also considered critical to
recovery.  Until studies are completed that provide a direct
comparison between these procedures done with and without
Carticel, it will not be possible to know exactly how much
Carticel contributes to repair of the knee. 
     FDA based its accelerated approval of Carticel primarily on
case-reports of 153 patients treated in Sweden.  Of patients who
were followed for at least 18 months after the treatment, about
70% showed improvement.   Also, biopsies were done on 22 patients
implanted with Carticel and 15 showed some development of hyaline
cartilage.  Hyaline cartilage allows the body to absorb shock and
friction and does not usually form after cartilage injuries. In
addition, over 1/2 of patients who failed to benefit from prior
surgical treatment without Carticel, had better, more long-lasting responses when Carticel was included.       
     Early data regarding knee function from a U.S. patient
registry treated with Carticel are consistent with those for the
Swedish study.      
     FDA grants accelerated or early approval to products that
treat serious or life-threatening illnesses when studies of the
products indicate early favorable outcomes that are likely to
predict clinical benefit.  However, companies are required to do
further studies to verify the clinical benefits.  
     In this case, the sponsor of Carticel, Genzyme Tissue
Repair, has committed to conducting two randomized controlled
studies.  One will help determine the contribution of the cells
to the treatment.  The other will compare long-term (at least 5
years) outcomes in patients treated with Carticel to outcomes
with other procedures currently in use. 
       Genzyme Tissue Repair is located in Cambridge,
Massachusetts.            
                         ####
[FDA HOME PAGE]