Amylyx Pharmaceuticals Inc. shares fell Friday after US regulators posted briefing documents that appeared to cast doubt on whether data on its experimental drug for amyotrophic lateral sclerosis would be sufficient to gain regulatory clearance.
(Bloomberg) — Amylyx Pharmaceuticals Inc. shares fell Friday after US regulators posted briefing documents that appeared to cast doubt on whether data on its experimental drug for amyotrophic lateral sclerosis would be sufficient to gain regulatory clearance.
The documents were published ahead of a reconvened meeting of Food and Drug Administration advisers next week, where the panel of experts will discuss and vote on whether to recommend approving the drug, called AMX0035. The shares fell as much as 33%, the most intraday since March 28, and were down 19% as of 1:45 p.m. in New York.
People with ALS, sometimes called Lou Gehrig’s disease after the former baseball star who suffered and died from it, live on average two to five years after their initial diagnosis. They often lose their ability to walk, eat and eventually breathe. There is no cure, and Amylyx is one of a few companies trying to fill the void.
The agency has been under fire for its handling of drug approvals for diseases like ALS that have few or no effective treatments. After its recent approval of Biogen Inc.’s Aduhelm for Alzheimer’s disease, the Centers for Medicare and Medicaid Services put significant restrictions on payment for the drug, citing a lack of persuasive data.
During an initial meeting of outside FDA advisors in March, the panel found that data were insufficient to show Amylyx’s treatment slows ALS progression and urged the agency to wait for more. However, the clinical trial is expected to complete in late 2023 or early 2024, with results available shortly thereafter.
This puts the agency in “a challenging situation of potentially making a regulatory decision that may not be subsequently aligned with the results of the ongoing study,” FDA staff said in briefing documents released Friday.
Amylyx is looking forward to presenting data to support the drug’s approval at next week’s meeting, according to a company statement
“We believe AMX0035 is an important potential new treatment in the fight against ALS, and we are working against the ALS clock and the ALS community does not have time to wait,” Amylyx co-Chief Executive Officers Justin Klee and Josh Cohen said in a statement.
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