US FDA panel backs accelerated approval for Biogen's ALS drug

[March 23, 2023]  By Bhanvi Satija

(Reuters) -A panel of advisers to the U.S. drug regulator voted that data on Biogen Inc's experimental treatment for ALS, also known as Lou Gehrig's disease, shows that the drug could work, potentially paving the way for an accelerated approval.

While the panel was divided on whether there was enough evidence to show the drug works, it voted in favor of data that showed the neurological disorder drug, tofersen, reduced levels of protein neurofilament in patients.

Neurofilament is considered a potential marker of nerve cell degeneration.

In a late-stage study, tofersen reduced levels of neurofilament, but failed to meet the main goal of reducing symptoms in patients with fast-progressing ALS.

The U.S. Food and Drug Administration (FDA) panel's vote was as expected and supportive of the drug's accelerated approval, said SVB Securities analyst Marc Goodman.

The FDA generally follows the advise of its experts, though it is not bound to do so.

In a second vote, the panel said there was not enough convincing evidence to show the effectiveness of tofersen for the disease, reducing the chances of a full approval for now.

A sign marks a Biogen facility in Cambridge, Massachusetts, U.S. January 26, 2017. REUTERS/Brian Snyder

"I think it meets the evidentiary standards for accelerated approval but not for full approval," said panelist Robert Alexander, a neurologist.

If tofersen is approved under the agency's accelerated approval pathway, a confirmatory trial will need to show the drug provides a clinical benefit for it to remain in the market.

ALS, or amyotrophic lateral sclerosis, leads to progressive paralysis and death. Its cause is largely unknown.

Biogen and partner Ionis are seeking approval to treat ALS patients with mutations in a specific gene that leads to toxic buildup of a protein known as superoxide dismutase 1.

(Reporting by Manas Mishra, Bhanvi Satija and Raghav Mahobe in Bengaluru; Editing by Shounak Dasgupta and Saumyadeb Chakrabarty)

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