FDA Granted Approval to Cenobamate, Risdiplam is Granted Priority Review Designation, Riluzole Oral Film FDA Approved for Treatment of ALS

This week's Neurology News Network covered the FDA approval for cenobamate, a drug used for the treatment of partial-onset seizures, as well as an FDA priority review for Risdiplam, and an FDA approval for an oral film of riluzole, a treatment for amyotrophic lateral sclerosis (ALS).

Marco: Welcome to Neurology News Network. I’m Marco Meglio. Let’s get into the news from this week.

The FDA has granted approval to cenobamate for the treatment of partial-onset seizures in adults. The approval was supported by data from pivotal trials that assessed the efficacy and safety of cenobamate in more than 1900 patients. It was shown in trials to significantly reduce partial-onset seizure frequency, with up to 20% of patients achieving seizure-free status during the maintenance phases. The therapy is expected to be available in the US in the second quarter of 2020, following scheduling review by the DEA, according to SK Life Science. The safety and efficacy of the sodium channel antagonist were established in 2 pivotal trials including 655 adults with partial-onset seizures with or without secondary generalization. Patients experienced these seizures for a mean of 24 years, with a median of 8.5 seizures per 28 days during the baseline period of 8 weeks.

The FDA has granted a priority review designation for the new drug application for risdiplam, an investigational and orally active small molecule therapy for the treatment of spinal muscular atrophy, according to an announcement from PTC Therapeutics. Risdiplam’s Prescription Drug User Fee Act date has been set for May 24, 2020. The NDA is supported by 12-month data from the dose-finding phases of the pivotal trials SUNFISH and FIREFISH. Notably, the filing acceptance by the FDA triggers a $15M milestone payable to PTC by Roche. This SMA development program is a collaboration between PTC, the SMA Foundation, and Roche.

The FDA has approved riluzole oral film for the treatment of amyotrophic lateral sclerosis (ALS). The approval is supported by several studies which demonstrated the drug’s bioequivalence to Rilutek, the liquid riluzole formulation marketed by Sanofi. Several riluzole formulations have come before the FDA in the last 2 years, with 7 approvals of the therapy worldwide. Most recently, in September 2018, ITF Pharma’s oral suspension, marketed as Tiglutick, was approved by the agency after being granted both fast track and orphan drug designations. That approval was the first easy-to-swallow thickened riluzole liquid for ALS.

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