ALS DRUG MYOTROPHIN 'POTENTIALLY APPROVABLE'
Company to Meet With FDA to Discuss Requirements
Myotrophin, an as-yet-unapproved, experimental treatment for amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) received a yellow light May 12 from the Food and Drug Administration. The drug companies Cephalon of West Chester, Pa., and Chiron of Emeryville, Calif., developers of Myotrophin, reported that the FDA said the drug is "potentially approvable, subject to submission of additional information from ongoing studies which demonstrates that Myotrophin is effective in the treatment of ALS."
The FDA's statement follows a May 1997 disapproval of Myotrophin by an agency subcommittee. An April 9 hearing on the subcommittee's recommendation was canceled.
ALS is a disorder of muscle-controlling nerve cells (motor neurons) that causes rapidly progressing paralysis and is ultimately fatal in almost all cases. Treatment is mostly supportive. One drug, riluzole (brand name Rilutek), is on the market as a specific treatment for the disease.
Officials of the drug companies said they plan to meet with the FDA "to clarify the conditions for approval and to determine whether these conditions can be satisfied." They said "there can be no assurance that the FDA's conditions for approval of Myotrophin can be satisfied by the companies at all or in a reasonable time frame."
Jason Rubin, a spokesman for Cephalon, said the FDA asked only for information from ongoing studies of the drug, including one in Japan. He said the agency did not ask the drug companies to conduct any new trials.
An FDA committee last year raised questions about Myotrophin's safety and effectiveness. In a large European trial of the medication, there were more deaths in Myotrophin-treated patients than in patients not taking the drug. In a similar trial in North America, Myotrophin-treated patients showed a slower progression of disease than untreated patients.
Myotrophin is based on a natural substance, insulin-like growth factor 1. It has to be injected.
Rubin said Myotrophin is still available to a limited number of people
with ALS via a random selection process. This "expanded access
program," which allows people to use an experimental drug, has
been in place for Myotrophin since mid-1996. To enter your name for
possible participation in the program or to follow developments regarding
Myotrophin, call Cephalon's information line at (800) 896-5855. The
company's Web site is www.cephalon.com.
-end-
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