Amylyx Pharma said yesterday the US Food and Drug Administration (FDA) approved its drug for slowing progression of ALS, or amyotrophic lateral sclerosis, and potentially delaying death, sending shares of the company up nearly 14 per cent in extended trading.
The company added healthcare professionals can soon write prescriptions for the drug, to be sold under the brand name Relyvrio, while it is working on the launch.
“We know people living with ALS have no time to wait, and, for this reason, Amylyx is prepared to move quickly with commercialisation in the US,” Josh Cohen and Justin Klee, Co-Chief Executive Officers, said in a statement.
Amylyx’s drug is a combination of generic compounds sodium phenylbutyrate and taurursodiol that work together to prevent nerve cells in the brain and spinal cord from dying prematurely.
Amylyx’s case for approval was built on a recent analysis of clinical trial data that it said showed the drug slows progression of the disease and extends life expectancy by nearly 10 months.
FDA staff had expressed concerns earlier this month that the additional analysis came from the same small mid-stage trial as initial data and could not be considered as confirmation of the drug’s benefits.
The FDA’s decision, a rare reversal, follows an approval recommendation from an outside panel of advisers. The agency reconvened the panel in September to look at the new data analyses after it had previously recommended against approving the drug in March.
Relyvrio is the third ALS drug to be approved by the FDA following Japanese firm Mitsubishi Tanabe’s Radicava and the generic drug riluzole.
EP News Bureau