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First Drug to Effectively Treat Disease Since it was First Described in 1869

COLLEGEVILLE, Pa. Dec. 12 /PRNewswire/ -- Rhone-Poulenc Rorer Inc. (NYSE: RPR) announced today that it has received clearance from the U.S. Food and Drug Administration (FDA) to market Rilutek® (riluzole), the first effective drug for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). No treatment was previously available for this fatal, neurodegenerative disease since it was first described 126 years ago.

Rilutek is the first drug shown to extend survival in people with ALS. The New Drug Application (NDA) received approval from the U.S. FDA less than six months after RPR submitted it on June 29, 1995.

"Rilutek is the first drug to demonstrate efficacy in the treatment of ALS," said Gary T. Shearman, Senior Vice President, Drug Development, RPR. "It represents a critical first step and important therapeutic gain in ALS treatment. Our number one goal is to make the drug available to ALS patients as quickly as possible."

Rilutek will be available by prescription in six weeks as a 50 mg tablet to be administered twice daily. An official launch news conference will be held on January 10, 1996, to discuss drug availability and patient support services (a separate announcement will identify the exact time and location). A toll-free telephone number will be available for people who cannot attend the conference.

People who have questions about Rilutek before January 10th can call 1-800-RX-TRIAL (1-800-798-7425) for more information.

Clinical Trial Results

In the largest ALS clinical trial ever, Rilutek was shown to extend the survival of ALS patients. A total of 959 patients participated in a multi-center, double-blind, placebo-controlled Phase III trial, in which survival served as the primary endpoint.

In addition to demonstrating a survival benefit, Rilutek was generally well-tolerated. Side effects reported in the trial included nausea, asthenia (fatigue) and elevated liver enzymes.

The Phase III trial was conducted at 31 sites in Europe and North America. In the United States, all centers were certified and funded by the Muscular Dystrophy Association. The participating centers were Baylor College of Medicine, Veterans Affairs Medical Center (Houston); Johns Hopkins University (Baltimore); Northwestern University (Chicago); California Pacific Medical Center (San Francisco); and Tufts-New England Medical Center (Boston).

ALS is a fatal, neuromuscular disease affecting approximately 25,000 to 30,000 people in the United States and 70,000 people worldwide. It attacks nerve cells in the brain and spinal cord, resulting in muscle paralysis and respiratory failure. Patients generally survive three to five years after diagnosis.

The outward signs of ALS are progressive weakness and deterioration of the muscles (amyotrophic), beginning in limbs, usually on one side of the body (lateral). Inside the body, the nerves controlling motor function die off after their cell bodies become hardened and shriveled (sclerosis), leaving the patient increasingly helpless.

The FDA allowed an Early Access Program (EAP) treatment protocol to proceed on June 20, 1995. The EAP was established by RPR to allow a limited number of people with ALS to receive Rilutek free-of-charge pending FDA approval and commercial availability. A total of 3,000 patients were selected to participate in the program.

Rhone-Poulenc Rorer is a global pharmaceutical company dedicated to improving human health.

WASHINGTON, Dec. 12 /PRNewswire/ -- The Food and Drug Administration today announced the approval of riluzole, the first drug that has been shown to prolong the survival of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

ALS, which results in progressive muscular weakness and paralysis, has been without cure since it was first identified in 1869. FDA carried out the review of riluzole in five-and-a-half months.

Commenting on the approval, Secretary of Health and Human Services Donna E. Shalala noted that when tested in two placebo-controlled studies on more than 1,000 patients, riluzole prolonged survival on average by about three months.

"This is a modest effect, but before riluzole there was no therapy for ALS at all," the Secretary said. "Today's approval is an important milestone in the decades-long search for treatment for this devastating disease."

FDA Commissioner David A. Kessler, M.D., pointed out that before today's approval, riluzole had been made available to more than 3,000 patients -- about one-tenth of the 30,000 Americans with ALS -- under an early access program granted by FDA and administered by the National Organization for Rare Disorders.

"This is the first drug we have ever had that seems to make a difference in the course of ALS -- a dreadful disease," Kessler said. "We hope this is just a first step."

ALS is a progressive disease that affects nerve cells in the brain and spinal cord and is usually fatal within five years after diagnosis. Adverse effects of riluzole observed in clinical tests include weakness, nausea, vomiting and elevated liver enzymes.

FDA's Peripheral and Central Nervous System Drugs Advisory Committee reviewed the data from trials on riluzole on September 18, and recommended its approval for treatment of ALS.

Riluzole is manufactured by Rhone-Poulenc Rorer Inc. of Collegeville, Pa., and is distributed under the trade name Rilutek.

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