Amylyx’s New Treatment for the ALS Patient Community: Lawrence Steinman, MD


The Zimmermann Professor of Neurology and Neurological Sciences and Pediatrics at Stanford University discusses what Amylyx’s FDA approved therapy means for the patient community and future research that may be performed regarding the dosage of the treatment. [WATCH TIME: 5 minutes]

WATCH TIME: 5 minutes

“My instinctive reaction is that they'll be successful in phase 3 and, in good conscience, I can recommend this drug in the meantime, of course.”

Recently approved by the FDA on September 29, 2022, was AMX0035 (Relyvrio; Amylyx Pharmaceuticals), a new treatment for patients with amyotrophic lateral sclerosis (ALS). The therapy is made up of a coformulation of sodium phenylbutyrate and taurursodiol, which attempts to reduce disease progression and lower the mortality risk.

The treatment became third approved for ALS based on its ability from findings in the phase 2 CENTAUR trial (NCT03127514), which was published in the New England Journal of Medicine. The trial was randomized and double-blinded, where 137 patients with ALS were included and were randomly assigned 2:1 to AMX0035 (3 g sodium phenylbutyrate and 1 g taurursodiol) or placebo. The patients received the treatment once per day over the course of 3 weeks, and then the dosage was twice increased per day over a 24-week period.1

In a recent conversation with NeurologyLive®, Lawrence Steinman, MD, Zimmermann Professor of Neurology and Neurological Sciences and Pediatrics, Stanford University, discussed the breakthrough of the approval of AMX0035 and what the treatment means for the ALS patient community. He also mentioned the implementation of a program for those who do not have insurance to access the drug, and future studies that may be conducted regarding the dosage of the therapy.

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
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