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December 30, 2021
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FDA accepts new drug application for ALS treatment

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The FDA has accepted a new drug application, or NDA, for a combination of sodium phenylbutyrate and taurursodiol intended to treat ALS, Amylyx Pharmaceuticals Inc. announced in a press release.

The FDA granted the treatment, called AMX0035, priority review and assigned it a prescription drug user fee act date of June 29, 2022. The agency said it intends to have an advisory committee meeting for the application.

“Our team is committed to bringing a potential new treatment, if approved, to people living with ALS as efficiently as possible, as each moment counts,” Joshua Cohen, co-CEO, chairman and co-founder of Amylyx, said in the release.

The submission rested upon data from the randomized, double-blind, placebo-controlled phase 2 clinical CENTAUR trial. The trial occurred across 25 centers of the Northeast ALS Consortium and included 137 individuals with ALS. Participants who received AMX0035 had statistically significantly reduced clinical decline based on the Revised ALS Functional Rating Scale at the end of the 6-month randomized phase.

A survival analysis included all randomly assigned participants from the CENTAUR trial who were followed for up to 3 years, including those who continued receiving AMX0035 in an open-label extension phase during follow-up. This analysis showed a 44% lower risk for death among those who started on AMX0035 in the placebo-controlled phase of CENTAUR compared with those who started on placebo in the placebo-controlled phase (HR = 0.56; 95% CI, 0.34-0.92).

Those who started on AMX0035 and those who started on placebo had median survival duration through the open-label long-term follow-up phase of 25 months (95% CI, 19-33.6) and 18.5 months (95% CI, 13.5-23.3), respectively.

The AMX0035 and placebo groups had similar overall reported rates of adverse events and discontinuations during the 24-week randomized phase; however, those in the AMX0035 group had higher rates of gastrointestinal events.

“There is a lot of progress in ALS research, and now with AMX0035’s NDA acceptance, we believe we are one step closer to a potential new treatment option,” Merit Cudkowicz, MD, co-principal investigator of the CENTAUR trial and co-founder of the Northeast ALS Consortium, said in the release. “We look forward to seeing AMX0035 potentially progress through the regulatory review process as we continue to investigate its therapeutic potential in the global phase 3 PHOENIX clinical trial as part of the collaboration with the Northeast ALS Consortium and the Treatment Research Initiative to Cure ALS in Europe.”