In ‘unusual’ move, FDA to reconvene advisers for ALS drug review

Dive Brief:

• Amylyx Pharmaceuticals said Tuesday that the Food and Drug Administration plans to reconvene a group of outside advisers to discuss an approval application for the company’s closely watched, experimental treatment for ALS.
• The FDA’s move is very unusual, according to Evercore ISI analysts Umer Raffat and Michael DiFiore. Though it’s commonplace for the agency to conduct advisory committee meetings when there are outstanding questions about a drug under review, each approval application almost always receives, at most, one of these meetings. The first for Amylyx’s drug, called AMX0035, took place in March, and now the second is scheduled for Sept. 7.
• Amylyx said the upcoming meeting will focus on additional data analyses from clinical trials of AMX0035 that have since been included in its application. In March, the FDA’s advisers narrowly voted against the drug, concluding that it shouldn’t be cleared for market until researchers have more evidence of its effectiveness.

Dive Insight:

ALS, short for amyotrophic lateral sclerosis, is a neurodegenerative disorder with few treatment options. In the U.S., two drugs are used to either alleviate symptoms of the disease or prolong survival — though both have limited effects. Patients currently live about two to five years, on average, after diagnosis.

AMX0035 could offer another option and, if approved, would be the first new ALS drug to enter the U.S. market in more than five years. A clinical study of around 140 patients found those who received its drug showed modest improvements in function and survival compared to those given placebos.

With that data in hand, Amylyx has, over the last year or so, asked regulators in Canada, Europe and the U.S. to approve its drug. Health Canada did so last month, allowing AMX0035 to be sold there under the brand name Albrioza. Amylyx has since been working through the reimbursement process in Canada, but said it expects its drug to become commercially available within six weeks of the approval.

Meanwhile, a verdict by FDA had also been slated for June. But the agency extended its review deadline to Sept. 29 to allow more time to assess a “major amendment” to Amylyx’s application, with the new information being additional clinical data.

Now, according to Amylyx, the FDA intends to bring back together its advisory committee for neurological drugs to take another look at AMX0035.

“We remain engaged with the FDA to advance AMX0035 through the review process as efficiently as possible,” said Tammy Sarnelli, Amylyx’s global head of regulatory affairs, in a statement.

“As we have heard from the ALS community, there is a crucial need for new and effective treatments in ALS, and our team will continue to work around the clock to advance treatments for ALS in the U.S.,” Sarnelli added.

The FDA’s request for a second meeting is rare, though, according to Raffat and DiFiore, the Evercore analysts. Aside from the repeat panels that the FDA held during the rapid development of COVID-19 vaccines, Raffat has “never encountered two [advisory committee meetings in the same] drug review.”

Yet, the two analysts have also argued that recent events and past commentary from FDA officials bodes well for approval of AMX0035. For example, across 156 cases in which the FDA held an advisory panel for a drug under review, Raffat and DiFiore found 23 where the approval decision deadline was extended. And among those, 18, or 78%, went on to be cleared for market.

Shares of Amylyx were up more than 10% late Tuesday morning, to trade at roughly $21.50 apiece.

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