Pharma Focus Asia

Aquestive Therapeutics Receives U.S. FDA Orphan Drug Designation for Riluzole Oral Soluble Film to Treat ALS

Thursday, February 01, 2018

Aquestive Therapeutics, Inc. today 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), a debilitating disease affecting as many as 30,000 Americans.i Riluzole OSF is currently in late-stage clinical development.

"We hope to bring riluzole OSF to ALS patients in the second half of 2019," said Keith J. Kendall, Chief Executive Officer of Aquestive. "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."

The FDA's Office of Orphan Products Development grants drug sponsors with orphan drug designation for medications that are intended for use in diseases which affect fewer than 200,000 persons in the U.S. This designation provides sponsors with opportunities for assistance with development of designated medicines for rare diseases, including eligibility for a seven-year period of market exclusivity in the U.S. following product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.

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