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Amyotrophic Lateral Sclerosis Drug AMX0035 (Relyvrio) Should Be Promptly Removed from the Market

WASHINGTON, D.C. — Amylyx Pharmaceuticals said on Friday that AMX0035 (sodium phenylbutyrate and taurursodiol, marketed as Relyvrio) did not provide any benefit to patients with amyotrophic lateral sclerosis (ALS) in a clinical trial of 664 subjects, and that it may voluntarily withdraw the drug from the market. In 2022, Public Citizen urged the Food and Drug Administration (FDA) not to grant AMX0035 approval because the data from a single phase 2 clinical trial of 137 subjects and its open-label extension study were inconclusive. Dr. Robert Steinbrook, director of Public Citizen’s Health Research Group, released the following statement.

“Although amyotrophic lateral sclerosis is a devastating disease and effective treatments are urgently needed, the FDA should never have approved AMX0035. If the manufacturer does not promptly remove the drug from the market, the FDA should require that the drug be removed.

“In approving AMX0035, the FDA capitulated to strong pressure from patients and advocates, many with ties to Big Pharma, and did not follow the science. There are lessons to be learned. Listening to patients and advocacy groups is important but has its limits, especially considering the frequent connections between advocacy groups and Big Pharma. The FDA must protect patients from medicines that don’t work by requiring convincing evidence of their clinical effectiveness before they are approved.”