FDA accepts new drug application to treat ALS

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December 30, 2021

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

The FDA gave the treatment a priority review, called AMX0035, and gave it a prescription drug user fee law date of June 29, 2022. The agency said it intends to organize an advisory committee meeting for the request.

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

The submission was based on data from the CENTAUR randomized, double-blind, placebo-controlled phase 2 clinical trial. The trial took place at 25 centers in the Northeast ALS Consortium and included 137 people 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 randomized participants in the CENTAUR trial who were followed up to 3 years, including those who continued to receive AMX0035 in an open-label extension phase during follow-up. This analysis showed a 44% lower risk of death in those who started treatment with AMX0035 in the placebo-controlled phase of CENTAUR compared to those who started with placebo in the placebo-controlled phase (HR = 0 , 56; 95% CI: 0.34-0.92). .

Those who started AMX0035 and those who started placebo had a median survival time during 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 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 the acceptance of the NDA of AMX0035, 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 statement. “We look forward to seeing AMX0035 potentially advance through the regulatory review process as we continue to investigate its therapeutic potential in the PHOENIX Phase 3 global clinical trial in collaboration with the Northeast ALS Consortium and Treatment Research Initiative to Cure ALS in Europe. “


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