Amylyx Pharma files for FDA approval of AMX0035 in ALS

US CNS-focussed drug biotech Amylyx Pharmaceuticals says it has now submitted a New Drug Application (NDA) to the US Food and Drug Administration for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine)) for the treatment of amyotrophic lateral sclerosis (ALS), often called Lou Gehrig's disease and motor neurone disease.

The NDA submission is based on data from the CENTAUR trial, a placebo-controlled study evaluating 137 people with ALS. In this study, participants receiving AMX0035 had statistically-significant slowing of functional decline at the end of the six-month randomized phase as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS.

The NDA filing this soon is seen as surprising as, back in April, the FDA told Amylyx it required more data from another placebo-controlled study, at which time the company drew up plans for that clinical trial and raised the money to finance it, in a $135 million serious C funding round.