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The US FDA approved Amylyx Pharmaceuticals (NASDAQ:AMLX) Relyvrio for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
The company’s shares are up 12% in after-hours trading.
Relyvrio (sodium phenylbutyrate/taurursodiol) is an oral drug in which the medication is dissolved in water. For the first three weeks, one packet is given daily. After this, the dosage is one packet twice daily.
Relyvrio was granted Priority Review designation as well as orphan drug status.
The approval was based on results of a single, placebo-controlled study of 137 adults with ALS. Those in the treatment group experienced a slower rate of decline on a clinical assessment of daily functioning compared to those receiving a placebo.
However, there were concerns about just how effective Relyvrio really is. Even the FDA conceded this in a summary memorandum accompanying the approval.
Regarding the study and the related analyses, the FDA wrote, there is “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; 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.”
Earlier the month, an agency advisory panel backed the drug by a vote of 7-2.
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