AMX0035 NDA Planned, gammaCore Approved to Treat Paroxysmal Hemicrania, Epilepsy Represents Risk Factor for SUD

This week Neurology News Network covered an announcement by Amylyx Pharmaceuticals regarding their plans to submit a NDA for AMX0035, as well as the FDA clearance for electroCore's gammaCore nVNS system in patients with paroxysmal hemicrania and hemicrania continua, and why epilepsy is a major risk factor for sudden unexplained death.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

Amylyx Pharmaceuticals has announced that it is prepared to submit a new drug application (NDA) to the FDA for its investigational agent AMX0035—a combination of sodium phenylbutyrate and taurursodiol (also known as ursodoxicoltaurine)—for the treatment of amyotrophic lateral sclerosis. This decision was made based on a recent discussion between the FDA and Amylyx, according to the company. Joshua Cohen, co-CEO, chairman, and cofounder, Amylyx, said in a statement that the company was “thrilled to move toward the US submission” and that they “look forward to continuing to work with the FDA.” The ALS Association lauded the news of Amylyx's decision to move forward with the submission of an NDA—marking its ongoing and longstanding support of the combination therapy. In June 2016, the association provided $750,000 in grant funding to Amylyx for a pilot trial of the therapy, following that up with a $1.46 million grant to the Northeast ALS Consortium (NEALS) to aid in funding the phase 2 clinical trial.

The FDA has cleared an expanded indication for electroCore’s gammaCore noninvasive vagus nerve stimulation (nVNS) to treat patients with paroxysmal hemicrania (PH) and hemicrania continua (HC) in adults, making it the first treatment—drug or device—to be approved for these patient populations. Data from multiple clinical audits and case series/case reports that included patients with PH or HC were the basis for the label expansion. Among a cohort of 14 patients with PH and 19 patients with HC, 79% of those experienced clinically meaningful benefits from the device for each indication. This included decreases in the severity of persistent pain and/or reductions in the frequency, severity, and/or duration of attacks. Notably, many patients reported at least 1 clinical benefit and no serious or unexpected adverse events occurred as well.

Findings from a nationwide retrospective population-based cohort study of Danish citizens aged less than 50 years old revealed that epilepsy is a major risk factor for sudden unexplained death (SUD), only second to cancer in adults and pneumonia in children. In addition, a lower sudden unexplained death in epilepsy (SUDEP) risk was observed in children compared to adults. The final dataset included 3,838,293 individuals, 30,437 (0.8%) of which had prevalent epilepsy, who were followed for 10,456,143 person-years (PY). Despite only representing 0.8% of the population, 700 of the 7825 recorded deaths were by persons with epilepsy, corresponding to 8.9% of all deaths. In comparison, 0.2% of persons without epilepsy died during the study period.

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