The FDA Monday approved Rhone-Poulenc Rorer Inc.'sRilutek (riluzole), which is the first drug to make it tomarket in any country for treatment of deadlyamyotrophic lateral sclerosis (ALS), also known as LouGehrig's disease.
Rilutek is a small molecule derived from a class ofcompounds called benzothiazoles. The drug is designedto decrease production of glutamate in the central nervoussystem. In excessive amounts, glutamate is linked todestruction of nerve cells and may be a cause of ALS.
About 70,000 people worldwide, more than half of whomare in the U.S., suffer from the neurodegenerative disease.ALS destroys motor neurons, leading to loss of musclecontrol and death from respiratory failure. It usually killsvictims in three to five years.
In clinical studies, Rilutek prolonged ALS patients' livesup to three months, but was not effective in slowingmuscle deterioration.
Rhone-Poulenc Rorer, of Collegeville, Pa., submitted itsnew drug application (NDA) for Rilutek in June 1995.For the past six months the company has made the drugavailable free to ALS patients worldwide under treatmentinvestigational new drug (IND) applications.
Bob Pearson, Rorer's spokesman, said that to date 3,000people in the U.S. have received the drug. The companywill begin charging for treatments when the Rilutektablets become commercially available in about sixweeks. The price will be disclosed Jan. 10, 1996.
Although Rilutek is not a cure, FDA CommissionerDavid Kessler said Monday, "This is the first drug wehave ever had that seems to make a difference in thecourse of ALS. We hope this is just a first step."
Another potential treatment, Cephalon Inc.'s Myotrophin,has shown in Phase III studies it also alters the course ofthe disease, slowing ALS progression and lessening itsseverity.
Jason Rubin, spokesman for Cephalon, said the WestChester, Pa.-based company is "currently awaitingapproval of our treatment IND and we're looking forwardto filing our NDA early next year."
Cephalon developed Myotrophin, which is humaninsulin-like growth factor, in a collaboration with ChironCorp., of Emeryville, Calif.
Some Wall Street analysts have speculated Rilutek andMyotrophin will be used together to treat ALS.
Rorer's stock (NYSE:RPR) closed Monday at $49.62,down 25 cents. Cephalon (NASDAQ:CEPH) ended theday at $30.50, up 50 cents. Chiron (NASDAQ:CHIR)was off $1.12 at $102. n
-- Charles Craig
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