FDA, Amylyx Meeting to Review AMX0035 for Possible Approval

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by Lindsey Shapiro, PhD |

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A U.S. Food and Drug Administration (FDA) advisory committee has scheduled a virtual meeting with Amylyx Pharmaceuticals to review its application for the approval of AMX0035 to treat amyotrophic lateral sclerosis (ALS), the company announced.

The online meeting to discuss data supporting the approval request is set for March 30.

Amylyx‘s announcement follows an FDA decision late last year to accept the application and place it under priority review, which reduces the review time from the standard 10 months to six months. An agency decision on AMX0035, which also has orphan drug status in the U.S., is expected by June 29.

Amylyx also filed for AMX0035’s approval in Canada and recently in Europe.

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“There are few treatments approved for ALS, a devastating disease that impacts a person’s ability to move, speak, eat and breathe,” Lahar Mehta, MD, head of global clinical development at Amylyx, said in a press release.

“We look forward to a robust scientific discussion with the members of the advisory committee panel regarding the clinical data submitted to support our New Drug Application for AMX0035,” Mehta added.

AMX0035 is a fixed-dose oral combination of sodium phenylbutyrate and tauroursodeoxycholic acid, molecules that work together to prevent nerve cell death. They do this by blocking stress signals within mitochondria, the energy production factories of cells, and the endoplasmic reticulum, a cell organelle is involved in protein production, modification, and transport.

Amylyx first applied for AMX0035’s U.S. approval in November 2021, with its request supported by data from the Phase 2/3 CENTAUR clinical trial (NCT03127514).

CENTAUR showed that 24 weeks of treatment with AMX0035 was safe and slowed functional decline relative to a placebo, with a trend toward a lower risk of hospitalization and prolonged survival in recently diagnosed patients with rapidly progressing ALS.

Most trial participants (92%) chose to enroll in an open-label extension study (NCT03488524) and receive AMX0035 for up to 30 months (about two and a half years).

Initially, the FDA requested data from a larger Phase 3 trial before considering the therapy for approval. But after further discussions, Amylyx announced the agency would review an application based on CENTAUR data alone.

The company launched that Phase 3 trial, called PHOENIX (NCT05021536), testing AMX0035 against a placebo for 48 weeks (about 11 months) in late October.

PHOENIX is currently enrolling up to 600 adults with ALS whose symptoms appeared within the previous two years, and has begun dosing. Its primary goal is to assess changes in functional decline, measured with the ALS Functional Rating Scale-Revised (ALSFRS-R), and in survival with treatment. Changes in lung function, the need for ventilation, and quality of life will also be monitored.

Eligible patients are expected to be enrolled at sites across the U.S. and Europe. Currently, only U.S. sites are listed on the trial document.

In partnership with the Canadian Neuromuscular Disease Registry (CNDR), Amylyx is gathering demographic, clinical, and functional outcome information on patients across Canada who are using AMX0035, with a goal of collecting the potential treatment’s first real-world data.