4/16/98
HEARING ON MYOTROPHIN CANCELED
FDA Clock Runs Out May 11
By Margaret Wahl
The scheduled April 9 Food and Drug Administration hearing on the experimental drug Myotrophin didn't take place for reasons that aren't entirely clear. The drug companies involved are seeking approval to market Myotrophin as a treatment for amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease), a rapidly progressive disorder in which muscle-controlling nerve cells die, causing paralysis and ultimately death.
According to an April 6 press release from the drug companies Cephalon (West Chester, Pa.) and Chiron (Emeryville, Calif.), makers of Myotrophin, the hearing was canceled "to allow the agency [FDA] to continue its review of the companies' new drug application (NDA) seeking clearance to market Myotrophin (mecasmerin) injection for the treatment of ALS."
Kori Beer, spokeswoman for Cephalon, made a statement by e-mail on April 7, saying: "All that we know is that the FDA has canceled the advisory committee hearing. It is our understanding that they were not prepared to make a presentation to the committee. Under the Prescription Drug User Fee Act, which establishes performance standards for the agency, the FDA has until May 12 to render a decision on the NDA. We're anxious to offer Myotrophin to all patients with ALS and hope the agency will approve it."
Susan Cruzan, spokeswoman for the FDA, said in an April 8 telephone interview that the hearing was postponed "because we haven't completed our analysis. Further analysis by the agency and the sponsor [Cephalon and Chiron] were not complete and ready for discussion. The FDA sets up meetings, but they are tentatively planned. They haven't completed the things they need to do for this; a lot goes into it."
The drug Myotrophin is based on a natural substance known as insulin-like growth factor 1, or IGF-1. It has to be injected. Animal and laboratory data have suggested that this substance might help preserve nerve or possibly muscle cells in people with ALS. However, the drug approval process for Myotrophin has not been smooth.
Cephalon and Chiron first went before the FDA in January 1996, at which time the agency turned down their application to distribute the drug to people with ALS in an "early access" program (a program of limited free distribution for a drug not yet approved for marketing). In June of that year, however, that application was approved, and the access program began shortly thereafter.
In May 1997, the drug companies again went before the FDA to try to get full marketing approval for the drug, submitting a New Drug Application, or NDA. This application was denied by a vote of 6 to 3 by the Peripheral and Central Nervous System Drugs Advisory Committee. The committee was especially concerned that, in a large European trial of Myotrophin, more patients died in the treated groups than in the untreated group. In a large North American trial, patients on Myotrophin fared somewhat better than untreated patients. The committee asked for more data to "break the tie" of the two contradictory trial results.
Following an outcry of protest from some members of the ALS community over the denial of the application for Myotrophin, the FDA announced it would make a final decision about the drug on Aug. 11, 1997. On that date, the decision was postponed until Nov. 11. That date also came and went, with the understanding that the FDA had to make a decision about Myotrophin within six months (May 11, 1998).
The final hearing was supposed to have taken place on April 9, but that meeting was also canceled. The clock runs out May 11.
You can keep up with developments regarding Myotrophin by calling Cephalon's
hot line at (800) 896-5855. If you press 1, you'll be told that, if
you want to enter the expanded access program for Myotrophin, you should
have your neurologist call the same number and start the enrollment
process. You or the neurologist can talk to an operator by pressing
3, and you can leave a voice mail message by pressing 2.
-end-
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