Compelling testimony from patients. Inconclusive benefit data. Accelerated path to a controversial approval for a long-awaited drug.
That was the scenario last year for Biogen’s flagging Alzheimer’s treatment Aduhelm—a process that the FDA has had to answer for and doesn’t want to repeat.
So it is with great consternation that the U.S. regulator is weighing Amylyx Pharmaceuticals’ application for its amyotrophic lateral sclerosis drug.
On Tuesday, the agency took the unusual step of announcing a second advisory committee meeting to discuss the merits and risks of AMX0035. The meeting is set for September 7 to assess additional analyses of data that was presented in March, which the FDA then concluded was “not exceptionally persuasive.”
“We are pleased that the members of the advisory panel will review additional analyses from our clinical studies, including recently published analyses, supporting the previously reported functional and overall survival benefit for AMX0035,” Amylyx’s Global Head of Regulatory Affairs Tammy Sarnelli said in a release
Investors also reacted positively to the news as Amylyx’s shares jumped 14% by late Tuesday morning.
Amylyx is conducting a phase 3 trial, dubbed Phoenix, but is hoping for an approval in the meantime. The new analysis is of a phase 2 open-label extension study, Centaur, which showed a slowing of disease progression in patients.
During the original advisory committee meeting, AMX0035 was voted down, 6-4, a blow to advocacy groups who urged for approval, citing a lack of treatment options for the fatal disorder, also known as Lou Gehrig’s disease. Patients have argued for the drug, saying they’re willing to accept the risk of taking it for the small chance that it might help.
“I too have friends with ALS. It’s a terrible disease," committee member Dr. Liana Apostolova, of the Indiana University School of Medicine, said after voting against the recommendation in March. "Just like Alzheimer’s, there is no cure for these disorders, and they affect not only patients but the whole family."
A month ago, with an FDA decision approaching in late June, the agency said it would extend the target date to September 29, giving Amylyx renewed hope for an approval.
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Since then, regulators in Canada have granted the drug a conditional approval. In that country, the drug is sold as Albrioza.