FDA approves controversial new drug designed to slow the progress of ALS
The Food and Drug Administration on Thursday approved a new drug to slow the progression of ALS, a fatal neurodegenerative disease for which there is no cure.
The Food and Drug Administration on Thursday approved a controversial new drug designed to slow the progress of Lou Gehrig's disease, a victory for patients and advocates despite limited evidence that the drug is effective.
The drug, from Amylyx Pharmaceuticals of Massachusetts, joins only a handful of drugs approved by the agency for the fatal neurodegenerative disease and its symptoms.
The disease, also called amyotrophic lateral sclerosis, or ALS, affects nerve cells needed for activities like walking, speaking and eating. There is no known cure, and most people live only two to five years after diagnosis, according to The ALS Association.
The FDA's decision was based on a single phase 2 clinical trial of 137 ALS patients that found people who took Amylyx’s drug, which will be sold under the name Relyvrio, lived about 10 months longer than those who didn’t. The drug also appeared to delay hospitalizations.
The medicine, which comes in a powder, is a combination of two existing products: sodium phenylbutyrate, which is prescribed to treat a metabolic disorder, and taurursodiol, an over-the-counter supplement used to help prevent liver disease.
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