The recently launched phase 2 HELIOS clinical trial from Amylyx Pharmaceuticals, aims to explore the potential of AMX0035 (sodium phenylbutyrate and taurursodiol) in treating Wolfram syndrome, a rare and complex genetic disorder. In a recent interview, Drs. Lahar Mehta and Fumihiko Urano discussed the AMX0035 Wolfram syndrome trial design, objectives and implications for future research…
Amylyx notches first global approval for oral ALS therapy Albrioza
Amylyx Pharmaceuticals (Nasdaq:AML) has received Health Canada approval for Albrioza (AMX0035). The FDA is anticipated to decide on Albrioza, which consists of sodium phenylbutyrate and ursodoxicoltaurine, by September 29, 2022. Amylyx is the first new therapy for ALS to win approval in Canada since 2018. The approval also is the first global regulatory approval for…
Amylyx Pharmaceuticals preps NDA filing for ALS drug AMX0035
Cambridge, Massachusetts–based Amylyx Pharmaceuticals intends to submit a New Drug Application (NDA) to FDA for AMX0035 (sodium phenylbutyrate and taurursodiol) to treat amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease. The company decided to file after a series of meetings with the FDA, including a pre-NDA meeting on July 15. AMX0035…
Amylyx Pharmaceuticals wins orphan drug designation from FDA
The neurodegenerative-diseased-focused biotech Amylyx Pharmaceuticals announced that it had received orphan drug designation for AMX0035, a proprietary blend of sodium phenylbutyrate and tauroursodeoxycholic acid. The Cambridge, Mass.-based company won that designation to treat Wolfram syndrome, a rare genetic disease associated with diabetes, progressive optic atrophy, hearing loss and severe neurological disabilities. The prognosis for patients…