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. ####