FDA Approves Riluzole Oral Film for Treatment of Amyotrophic Lateral Sclerosis

Riluzole oral film (Exservan, Aquestive Therapeutics) has received early-action approval from the FDA for the treatment of amyotrophic lateral sclerosis (ALS), according to a company press release.

“We received full FDA approval for Exservan in advance of our PDUFA action date. We appreciate the ongoing feedback from the FDA and its early-action approval. We anticipate that Exservan, via our orally administered PharmFilm® dosage form, will bring meaningful treatment to patients who are diagnosed with ALS and face difficulties swallowing or administering traditional forms of medication,” said Keith J. Kendall, CEO of Aquestive, in a statement.¹

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. The progressive degeneration of the motor neurons in ALS eventually leads to their demise, and when these motor neurons die, the ability of the brain to initiate and control muscle movement is lost.²

Riluzole was the first treatment approved by the FDA to treat ALS and is an oral formulation that works to slow the progression of ALS symptoms and prolong survival

The first clinical trial evaluating riluzole was a randomized, double-blind clinical study that followed 155 patients with ALS in France and Belgium for 13 to 18 months. The participants were randomly assigned to receive either 50 mg of riluzole or a placebo twice daily. The results demonstrated that riluzole had a significant effect on the rate of survival.³

The second trial tested the effect of different doses of riluzole. A total of 959 patients with ALS from Europe and North America were randomly assigned to receive either 50, 100, or 200 mg of riluzole or a placebo per day for 12 months. There was no difference observed between the group taking the placebo and the patients given 50 mg a day of riluzole. However, the researchers saw an early increase in survival rates in patients treated with either 100 or 200 mg a day of riluzole compared with a placebo. The median survival time was about 60 days longer in the riluzole-treated group than in the placebo-treated group.⁴

The most recent study, published in The Lancet Neurology, re-examined the patient data from the second trial and found that riluzole prolongs survival in the last clinical stage of ALS, although more research is needed to confirm this finding.⁵

Riluzole oral film is now approved for the treatment of ALS. According to the press release, the oral film formulation of riluzole will now fill a critical need in the armamentarium for patients with ALS because it can be administered safely and easily, twice daily, without water, where many patients have trouble swallowing.¹

Reference

• Aquestive Therapeutics Receives FDA Approval for Exservan™ (riluzole) Oral Film [press release]. Globe Newswire website. Warren, NJ. Published November 25, 2019. http://aquestive.com/aquestive-therapeutics-receives-fda-approval-for-exservan-riluzole-oral-film/. Accessed November 25, 2019.
• What is ALS? ALS Association. http://www.alsa.org/about-als/what-is-als.html. Accessed November 25, 2019.
• Bensimon G, Lacomblez L, Meininger V, et al. A controlled trial of riluzole in amyotrophic lateral sclerosis. New England Journal of Medicine. 1994. Doi: 10.1056/NEJM199403033300901.
• Lacomblez L, Bensimon G, Meininger V, et al. Dose-ranging study of riluzole in amyotrophic lateral sclerosis. The Lancet. 1996. Doi: https://doi.org/10.1016/S0140-6736(96)91680-35
• Fang T, Khleifat AA, Meurgey JH, Jones A, et al. Stage at which riluzole treatment prolongs survival in patients with amyotrophic lateral sclerosis: a retrospective analysis of data from a dose-ranging study. The Lancet Neurology. 2018. Doi: https://doi.org/10.1016/S1474-4422(18)30054-1.