Amyotrophic lateral sclerosis (ALS) remains a bewildering disease. Although it is rare, it is not uncommon. The lifetime risk of developing the condition is roughly 1 in 300 by age 85. The heterogeneity of the disease is also unique, said Tammy Pighin Moore, CEO of the ALS Society of Canada. “About 5% to 10% of the people diagnosed…
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…