The FDA approved Rhone-Poulenc Rorer Inc.'s request to make itsdrug for Lou Gehrig's disease available to patients on a limited basispending the agency's decision on the company's marketingapplication.
Rhone-Poulenc Rorer, of Collegeville, Pa., said its early accessprogram for amyotrophic lateral sclerosis (ALS) patients will beginnext month and the drug, Rilutek, or riluzole, will be provided free ofcharge on a lottery basis.
An FDA spokeswoman said the Rhone-Poulenc request wasapproved under a treatment investigational new drug (IND)application, which provides for the use of drugs for life-threateningand seriously debilitating diseases prior to market approval. Since1987 when the FDA started granting treatment INDs, about 35 havebeen approved.
For a drug candidate to be eligible for such limited distribution, RobPartridge, spokesman for Rhone-Poulenc, said it must be safe andmust have demonstrated some evidence of benefit. In addition, therecan be no other treatment available for the patients.
ALS is a degenerative neuromuscular disease that destroys motorneurons, leading to loss of muscle control and death from respiratoryfailure. An estimated 70,000 people worldwide suffer from thedisorder and half are in the U.S. With no cure or effective treatmentsavailable, patients die three to five years after the disease strikes.
Rilutek, a small molecule compound, is the first ALS drug to bemade available to patients outside clinical trials. Partridge saidRhone-Poulenc Rorer plans to file for marketing approval in the U.S.and worldwide in July.
Earlier this month, Cephalon Inc., of West Chester, Pa., reportedpositive Phase III trial data showing its ALS drug, Myotrophin,slowed progression of the disease and improved patients' conditions.Myotrophin is a recombinant human insulin-like growth factor. (SeeBioWorld Today, June 12-13, p. 1.)
Cephalon expects to file its new drug application in the U.S. by early1996. A second Phase III study in Europe is expected to becompleted next month.
Rhone-Poulenc Rorer released results of its Phase III study of Rilutekin May. The data demonstrated the drug prolonged patients' lives upto three months, but was not effective in slowing muscle functiondeterioration. (See BioWorld Today, May 11, p. 1.)
Some Wall Street analysts have speculated that the drugs will be usedtogether if they are both approved by regulatory authorities.
Under the treatment IND, Rhone-Poulenc will make Rilutek availableto 1,000 patients a month in the U.S. on a lottery basis. The drug alsowill be provided to patients in other countries as their governmentsapprove early access programs.
Partridge said that by the end of the year Rhone Poulenc, which is asubsidiary of France-based Rhone-Poulenc Group, will have enoughdrug manufactured to treat 10,000 patients worldwide. Rilutek will bedistributed free until it receives market approval. The company hasnot determined a proposed price for the drug when it'scommercialized.
In the U.S., the treatment IND will be run by the NationalOrganization for Rare Disorders, which is a collection of non-profithealth organizations headquartered in Fairfield, Conn. ALS patientscan telephone (800) 798-7425 to participate.
Names of the first 1,000 people will be drawn at random July 24,Partridge said. In each succeeding month, another 1,000 names willbe selected.
Partridge said the program gives Rhone-Poulenc an opportunity toacquaint physicians with Rilutek as well as continue to collect safetydata on the drug.
Rhone-Poulenc's stock (NYSE:RPR) closed Thursday at $41.75, up87 cents. Cephalon (NASDAQ:CEPH) gained $1.37 to end the day at$18.75. n
-- Charles Craig Staff Writer
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