USA—Amylyx Pharmaceuticals Inc. has announced the initiation of the process to withdraw its drug, known as Relyvrio in the United States and Albrioza in Canada, used for the treatment of amyotrophic lateral sclerosis (ALS), effective immediately.

The decision to remove the drug from the market was based on topline results from the PHOENIX trial, a phase 3 randomized, double-blind, placebo-controlled study conducted at 65 sites across the U.S. and Europe, evaluating the safety and efficacy of Relyvrio in 664 adults with the neuromuscular condition.

This decision comes shortly after a clinical trial revealed that the once-promising treatment for the fatal disease is ineffective.

The primary efficacy outcome of the study was the change from baseline in the ALS Functional Rating Scale-Revised total score at 48 weeks.

Despite demonstrating favorable safety and tolerability profiles, results from the study indicated that Relyvrio did not outperform placebo on the functional rating scale.

The decision by Amylyx Pharmaceuticals to voluntarily withdraw Relyvrio from the market is uncommon but fulfills a pledge made by its executives in 2022 during the FDA evaluation process

The oral, fixed-dose combination therapy, also known as AMX0035, received FDA approval in September 2022.

However, the drug’s approval sparked controversy due to being based on a single clinical trial, with FDA staff and outside experts questioning its effectiveness.

Typically, the FDA requires more evidence, including two trials providing compelling data, to approve a drug.

However, it also exercises flexibility, considering factors such as the seriousness of the disease and the adequacy of existing treatments.

Amylyx stated its intention to continue collecting available data on survival at the encouragement of ALS specialists, while allowing the trial’s open-label extension to proceed.

Following the withdrawal, Relyvrio will not be available to new patients. However, current patients prescribed the drug who wish to continue treatment, in consultation with their physician, will be transitioned to a free drug program.

“Although this is a challenging moment for the ALS community, we have arrived at this decision in collaboration with the stakeholders affected and in line with our unwavering commitment to individuals living with ALS and other neurodegenerative diseases,” said Amylyx co-CEOs Joshua Cohen and Justin Klee in the release.

Amylyx announced plans to advance programs involving AMX0035 in Wolfram syndrome and progressive supranuclear palsy, as well as investigate AMX0114, an antisense oligonucleotide targeting calpain-2, for ALS.

In response to this development, Amylyx announced a restructuring to focus the company’s financial resources on upcoming clinical milestones. This includes a reduction of approximately 70% in its workforce and a decrease in external financial commitments outside of its priority areas.

Approximately 6,000 people in the United States are diagnosed with ALS each year, commonly referred to as Lou Gehrig’s disease, which progressively impairs the body’s ability to move, speak, and eventually breathe. There is currently no known cure for ALS.

Sales of Relyvrio generated approximately $380 million in revenue last year, and at one point, Amylyx had a market capitalization exceeding $1 billion.

Despite this setback, Amylyx has committed to studying the results of its larger trial to inform future ALS research.

The company continues to develop another ALS drug, although its leading drug candidate is currently for Wolfram syndrome, a hereditary disorder that causes diabetes.

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