Aquestive’s new drug application for Exservan — an investigational oral film of riluzole to be used as an add-on therapy for amyotrophic lateral sclerosis (ALS) — has been accepted by the U.S. Food and Drug Administration (FDA), with a final decision on its approval expected before the end of the year.
The regulatory authority now will start reviewing the new riluzole formulation, consisting of strips that dissolve over the tongue without the need for water. A final FDA decision on whether Exservan is approved or not for sale, is expected by Nov. 30, according to the Prescription Drug User Fee Act (PDUFA) goal date, the company said in a press release.
The therapy’s formulation is designed to make it easier to take for patients with difficulty swallowing, a common symptom of ALS.
Riluzole, Exervan’s active ingredient, was the first medicine approved by the FDA to treat ALS, in December 1995. It belongs to a class of medications called benzothiazoles and works by changing the activity of certain natural substances in the body that affect nerves and muscles. In ALS, it slows symptom progression and prolongs survival.
Exservan is a riluzole-containing film that is placed over the tongue (lingual delivery). The film quickly dissolves and releases the riluzole, which is absorbed by the gastrointestinal tract in much the same way as taking a tablet or a capsule. Previous patient studies showed Exservan can be taken safely and easily, twice daily, without water.
Prior clinical trials have demonstrated the therapy is safely swallowed by patients and has an equivalent pharmacokinetics profile — absorption, availability, distribution, metabolism, and elimination by the body — to Rilutek.
The new formulation makes use of Aquestive’s PharmFilm technology, which combines permeation enhancers, stabilizers, and polymer blends to ensure steady delivery of the therapy throughout the lining of the mouth. That should lead to more consistent and accurate dosing, making it easier for patients to adhere to therapy, according to the company.
Riluzole oral film (ROF) gained FDA orphan drug status in January 2018.
“We’re pleased the FDA has accepted our NDA for Exservan, given the needs ALS patients have for treatment advances. ALS, though relatively rare, represents a considerable economic and social burden in the U.S. and globally,” said Daniel Barber, Aquestive’s chief strategy and development officer.
“Oral films offer great promise to patients who face difficulty swallowing or administering traditional forms of medication. … We believe these innovations are important and can help patients interact consistently and compliantly with their medication,” he added.