The Food and Drug Administration (FDA) yesterday approved a controversial new drug designed to slow the progress of amyotrophic lateral sclerosis (ALS), a victory for patients and advocates despite limited evidence that the drug is effective.
The drug, from Amylyx Pharmaceuticals of Massachusetts, joins the few drugs approved by the agency for this disease fatal neurodegenerative disease and its symptoms.
Last July, Biogen’s new drug application was accepted as a treatment for ALS related to SOD1 mutations and last week the data of a drug, still in phase, were published in “The New England Journal of Medicine”. of research and developed to treat a rare and hereditary form of amyotrophic lateral sclerosis (ALS), which was able to reduce the molecular signs of this disease, although after six months the drug did not improve motor control or muscle strength.
ALS affects the nerve cells needed for activities such as walking, talking, and eating. There is no known cure, and most people live only two to five years after diagnosis.
Its price may be similar to that of an older ALS drug, edaravone, which costs about $170,000 a year.
The FDA’s decision was based on a single phase 2 clinical trial involving 137 ALS patients that found that people who took the drug, to be sold under the name Relyvrio, lived about 10 months longer than those who didn’t. The drug also seemed to delay hospitalizations.
The drug, which comes in powder form, is a combination of two existing products: sodium Phenylbutyrate, prescribed to treat a metabolic disorder, and the taurorsodiolan over-the-counter supplement used to help prevent liver disease.
Amylyx has said it is still calculating what it will charge for the drug. Its price may be similar to that of an older ALS drug, edaravone, which costs about $170,000 a year, according to a report from the Institute for Clinical and Economic Review.
However, the approval is more than likely to cause some disagreement among neurologists who treat ALS.
The FDA generally requires at least two well-controlled clinical trials to show that a drug is effective or a single trial that is “statistically highly persuasive,” Holly Fernandez Lynch, associate professor of medical ethics at the University of Pennsylvania, told NBCNews. Amylyx’s essay, she said, did not meet agency standards.
Doubts about the trial results arose in March, when the drug was first presented to an FDA advisory committee. In briefing papers released before the meeting, agency scientists questioned the Amylyx trial. The committee narrowly voted against recommending approval.
However, the FDA took the unusual step of convening a second advisory committee only six months later, after Amylyx submitted additional analysis of its trial data. At that meeting, the advisory committee changed course and voted to recommend the drug.