FDA approves Amylyx ALS drug

The Food and Drug Administration has greenlit Relyvrio, a drug produced by Amylyx Pharmaceuticals to treat adults with amyotrophic lateral sclerosis, or ALS. 

ALS, commonly referred to as Lou Gehrig's Disease, is a relentlessly progressive and fatal neurodegenerative disorder that affects at least 200,000 people worldwide, including approximately 29,000 Americans.

ALS is caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually death. The current median survival of people with ALS is less than 3 years from symptom onset and less than 2 years from diagnosis.

More than 90% of people with ALS have sporadic disease, showing no clear family history. 

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Amylyx Co-CEOs Joshua Cohen and Justin Klee called Relyvrio's approval from the FDA an "exciting milestone" for the ALS community and a "major step" toward ending the suffering caused by neurodegenerative diseases.

"Our priority now is to ensure that adults living with ALS in the U.S. whose doctors have prescribed Relyvrio can access it as quickly as possible," Amylyx Chief Commercial Officer Margaret Olinger added. "Physicians will be able to prescribe immediately, and we anticipate specialty pharmacies will be able to start to fill prescriptions and ship Relyvrio to people with ALS in the next four to six weeks."

Relyvrio costs around $12,500 per 28-day prescription, resulting in a wholesale cost of $158,000 per year. Amylyx plans to provide the drug at no cost to uninsured or underinsured ALS patients in the U.S. if they meet certain financial eligibility criteria and have exhausted all other options.

Relyvrio's approval was based on data from a 6-month randomized, placebo-controlled clinical trial involving 137 participants living with ALS. The Phase 2 study, which was funded in part by the ALS Ice Bucket Challenge, found that Relyvrio significantly slowed the loss of physical function in people living with ALS. 

The most common adverse effects with Relyvrio included diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Gastrointestinal-related adverse reactions occurred throughout the study but were more frequent during the first three weeks of treatment.

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The approval comes despite a "degree of residual uncertainty" from the FDA about the evidence of the drug's effectiveness. 

"There are limitations to these findings that result in a degree of residual uncertainty about the evidence of effectiveness that exceeds that which might typically remain following a conclusion that substantial evidence of effectiveness has been demonstrated," the FDA wrote in a summary memorandum. "However, given the serious and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this instance and consideration of these results in the context of regulatory flexibility is appropriate."

Relyvrio is the third ALS drug to be approved by the FDA after Japanese firm Mitsubishi Tanabe's Radicava and the generic drug riluzole.