Amylyx’s ALS drug gets approved in Canada

Dive Brief:

• The Canadian drugs regulator has given the go-ahead to a closely watched treatment for ALS, marking a milestone for the medicine’s developer Amylyx Pharmaceuticals as well as patients, who continue to have limited treatment options.
• Health Canada based its decision on results from a study of about 140 patients with rapidly progressing ALS, or amyotrophic lateral sclerosis, which showed the drug offered modest benefits on survival and day-to-day function. The approval, though, comes with some added requirements, including the submission of data from a much larger clinical trial expected to produce results in 2024.
• In the meantime, Amylyx will be launching its first product. The Massachusetts-based company expects its drug, which will be sold as Albrioza, will be commercially available in Canada within the next six weeks. Estimates hold that roughly 3,000 people in Canada are living with ALS.

Dive Insight:

Amylyx took a slightly unusual path to getting its first drug cleared for market.

Often, a pharmaceutical company will first ask for approval in the U.S., especially if that’s where much of the clinical testing used to support its case took place. But with Albrioza, Amylyx chose to file in Canada before anywhere else.

Josh Cohen, one of the company’s co-founders and co-CEOs, told BioPharma Dive last year that his team made this choice because of how receptive Health Canada regulators were to the Albrioza application. “They moved really quickly with us,” Cohen said in a March 2021 interview, adding then that the Food and Drug Administration “is at that point now. But it took them a little bit to sort of come around.”

Indeed, the FDA has appeared more cautious on Albrioza. For example, Amylyx revealed last April how the agency didn’t see its roughly 140-person study as persuasive enough to support an approval application, and recommended the company run another placebo-controlled trial before filing.

But, facing intense criticism from patient groups, the FDA ultimately back-tracked and encouraged Amylyx to seek approval while simultaneously running that additional trial. The company filed its application in November, and by late December, the FDA had agreed to review it.

Whether the agency follows Health Canada’s lead is unclear, however. In March, a panel of experts who advise the FDA on new brain drugs voted by a slim margin against approving Albrioza, which was previously known as AMX0035. Though the FDA isn’t bound to follow the recommendations of its advisors, it typically does.

The agency also recently pushed back the deadline for issuing a verdict on Albrioza. According to Amylyx, the delay stems from the submission of additional clinical data that the FDA must review. An approval decision, which was slated for late June, is now expected by Sept. 29.

On Wall Street, analysts have been torn about how to view these developments. Marc Goodman of the investment firm SVB Securities recently wrote to clients how, following the “disappointing” advisory panel vote, “the investment community has expected AMX0035 to not get approved by the FDA.”

And in a Monday note, Goodman argued the Canadian approval offers “no direct read-through into the FDA’s upcoming final decision in September.” He wrote, too, how the “base case scenario” is that Amylyx will need positive results from that larger, ongoing study in order to secure approval in the U.S.

Still, others have adopted a more positive outlook. Evercore ISI analysts Michael DiFiore and Umer Raffat recently analyzed 156 cases where FDA held an advisory panel for a drug under review, for example. They found 23 for which the agency extended its review deadline, and among those, 18 — or 78% — were later approved.

That track record “bodes incrementally-well,” for Amylyx and its drug, the analysts wrote in a June 3 note to clients.

Amylyx’s share priced opened at $13.09 Monday, down 3% from Friday’s market close. The company’s shares did tick up, though, to trade higher by late morning.

An Amylyx spokesperson confirmed the company is not disclosing the list price for Albioza, as it works through the standard Canadian reimbursement process.

Currently, just two drugs are used in the treatment of ALS, and both have their limitations. There remains no cure for the neurodegenerative disease, which is typically fatal within three to five years after patients start to show symptoms.