Breakthrough in ALS With FDA Approval of AMX0035: Lawrence Steinman, MD

WATCH TIME: 5 minutes

“I think there is a very intelligent and strategic policy at the FDA to give a so-called expedited approval, conditional upon actually doing a phase 3 [trial], but in the meantime, it's available to patients. I think that's the most important thing.”

The FDA approved AMX0035 (Relyvrio; Amylyx Pharmaceuticals), a coformulation of sodium phenylbutytrate and taurursodiol, on September 29, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). AMX0035 was approved based on findings from the phase 2 CENTAUR trial (NCT03127514) and is a treatment to help slow the progression and mortality of the disease.

Data from the phase 2, randomized, double-blind, CENTAUR trial (NCT03127514), supported the new drug application for AMX0035, which was submitted in November 2021. The data were published in the New England Journal of Medicine and included 137 patients with ALS who were randomly assigned 2:1 to AMX0035 (3 g sodium phenylbutyrate and 1 g taurursodiol) administered once per day for 3 weeks and then twice per day, or placebo, over a 24-week treatment period.¹

In a recent conversation with NeurologyLive^®, Lawrence Steinman, MD, Zimmermann Professor of Neurology and Neurological Sciences and Pediatrics, Stanford University, discussed the approval of AMX0035 and what patients and the healthcare industry still need for even more effective and safer breakthroughs for the disease treatment. He mentioned that it is a great breakthrough in science and believes that it will be successful in the phase 3 trial in the upcoming future.

REFERENCES
1. Paganoni S, Macklin EA, Hendrix S, et al. Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med. 2020;383:919-930. doi:10.1056/NEJMoa1916945