New Riluzole Formulation Gets Orphan Drug Designation for ALS


This morning, Aquestive Therapeutics, Inc. announced that the U.S. FDA has granted orphan drug designation to Riluzole Oral Soluble Film (riluzole OSF) for the treatment of ALS.

This morning, Aquestive Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Riluzole Oral Soluble Film (riluzole OSF) for the treatment of amyotrophic lateral sclerosis (ALS).

In December 1995, riluzole, then developed by Rhone Poulenec Rorer and marketed as Rilutek, received its initial approval because it exhibited the ability to provide clinical benefit to ALS patients. In what was, at the time, the largest ALS clinical trial ever, the drug was proven to extend the survival of patients by 3 months.

ALS, or "Lou Gehrig's Disease," is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The condition is characterized by neurological symptoms, including: seizures, delayed motor development, and cognitive problems that often develop from a cholesterol buildup in the brain.

"We hope to bring riluzole OSF to ALS patients in the second half of 2019," said Keith J. Kendall, Chief Executive Officer of Aquestive in a press release. "Patients suffering from this debilitating neurodegenerative disease often find swallowing to be difficult or impossible. To help manage one element of this challenging disease for people with ALS and their caregivers, we developed riluzole OSF to dissolve instantly in the mouth without water, using our proprietary PharmFilm® technology."

Riluzole is developed to preferentially block Tetrodotoxin (TTX)-sensitive sodium channels, which are associated with damaged neurons. It has also been seen to directly inhibit the kainite and N-methyl-D-aspartate (NMDA) receptors. It is currently available in a tablet and liquid form, the latter of which caters to the segment of the patient population with swallowing difficulties.

The recommended dose of Riluzole is one 50-mg tablet every 12 hours. It is suggested that the drug be taken on a regular basis and at the same time of day, every day, at least 1 hour before or 2 hours after a meal.

Reported side effects of the drug have included: nausea, stomach pain, low fever, loss of appetite, dark urine, clay-colored stools, jaundice, fever, chills, body aches, flu-like symptoms, chest discomfort, dry cough, trouble breathing, and others.

Riluzole has also been studied as a potential treatment for spinal muscular atrophy (SMA), however, efficacy was not observed.

A study conducted by a team of researchers at the Vitry-Alfortville Research Centre in Vitry-sur-Seine, France and published by the National Institute of Health (NIH) in 1996 evaluated the pharmacology and mechanism of action of Riluzole.

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