Tag Archive for: AMX0035

Amylyx Pharmaceuticals on Thursday released interim results from its ongoing Phase II HELIOS trial, showing that its lead asset AMX0035 (sodium phenylbutyrate and taurursodiol) improved pancreatic function and glycemic control in adult patients with the rare, inherited neurodegenerative disease Wolfram syndrome.

The agency’s top five represent an eclectic mix of cancer, cardiovascular and rare disease drugs.

Following a favorable vote from an FDA advisory committee, Amylyx is one step closer to seeing its amyotrophic lateral sclerosis (ALS) treatment approved for use in the United States.

Wednesday offers a rare opportunity for Amylyx Pharmaceuticals, which will go before the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee in a second attempt to win recommendation for its amyotrophic lateral sclerosis (ALS) drug, AMX0035.

The agency will reconvene a meeting of its outside experts to review the company’s application seeking approval for its amyotrophic lateral sclerosis (ALS) treatment.

Canada has approved Amylyx Pharmaceuticals’ treatment for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease, the first such approval for the oral drug. The decision came with certain conditions, including the release of data from its global late-stage study of about 600 patients, which is expected in 2024, as well as additional studies.