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Home > BREAKING NEWS: FDA Votes to Approve AMX0035 Treatment for ALS

BREAKING NEWS: FDA Votes to Approve AMX0035 Treatment for ALS

by | Sep 29, 2022

In an amazing update for the ALS community, on September 29, 2022, the FDA voted to approve ALS drug AMX0035 for the treatment of ALS. This comes a year after Amylyx, a Massachusetts-based pharmaceutical company, submitted a New Drug Application (NDA) for AMX0035, a drug that has shown to slow the progression of ALS and extend life for those with the disease by around 6.5 months.

Here’s everything you need to know about how we got here and what this means for the ALS community:

Identifying the Benefits of AMX0035

The AMX0035 treatment combines two compounds called sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). The results of the phase 2 trial (also known as CENTAUR) were published in September 2020. This study was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium and included 137 people living with ALS.

The results showed that people living with ALS experienced a significantly slower decline from disease progression compared to those on a placebo. And more importantly, the study provided data that showed drug recipients lived about 6.5 months longer than those who received a placebo. The CENTAUR trial also showed the drug to be safe and well-tolerated demonstrating a positive benefit and risk consideration for people with ALS.

Following this discovering the ALS Association and I AM ALS began campaigning for the FDA to move with urgency to ensure this treatment became available for the ALS community without requiring a Phase 3 trial.

To further show their commitment, the ALS Association committed $750,000 to Amylyx for research funding and $1.4M to the NEALS clinical trial consortium to support the trials. This was made possible by the countless supporters during the 2014 Ice Bucket Challenge, making AMX0035 the first ALS treatments to become FDA approved that was funded by the initiative.

In 2021, they began launching a new 48-week Phase three trial (aka PHOENIX). The PHOENIX trial aimed to enroll 600 participants across 55 sites, making it the first collaborative effort between TRICALS (Europe) and NEALS (US).

Just over a year following the release of the CENTAUR trial results, Amylyx submitted a New Drug Application for AMX0035 in October 2021. The company had previously filed approval in Canada and the European Union. In December that year the FDA accepted the application for review, granting it priority review. The administration gave an initial decision deadline of June 29, 2022.

Roadblocks on the Pathway to Access

In March 2022, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted against recommending the drug. The committee perceived that there was not sufficient evidence to prove the treatment effective. This was devastating news for those interested in the treatment. Historically, the FDA does not vote opposite of their advisory committees, but hope was not lost yet as the FDA had not announced their final decision.

As the June approached, ALS advocates wrote many letters urging the FDA to swiftly approve AMX0035. The FDA would announce in the beginning of June that they were extending the review deadline till September 29, giving them three more months to evaluate additional data and testimonies.

This same month, Health Canada approved AMX0035 as ALBRIOZA, under a Notice of Compliance with Conditions pathway. One of those conditions is that the PHOENIX trial confirms safety and efficacy of the treatment.

What changed the tide was that AMX0035 was granted a rare second advisory committee review in September 2022, just over three weeks before the new review deadline. During this meeting the Peripheral and Central Nervous System Drugs Advisory Committee reviewed additional data submitted by Amylyx which showed additional survival data. This information proved more reassuring to the advisory committee causing them to vote 7-2 in favor of recommending the drug be approved.

On September 29, 2022, the FDA officially voted to approve of AMX0035 for the treatment of ALS, making this the third FDA approved treatment for the disease.

The results of the PHOENIX trial will still impact the possibilities for this treatment. Co-Chief Executive Officer, Justin Klee shared that if the trial is unsuccessful, Amylyx will voluntarily remove AMX0035 from the market. The ALS Association will follow these developments closely.

What Comes Next?

This is a historic milestone made possible through the collaborative effort of ALS organizations and countless advocates across the country. We thank you for your commitment to the fight against ALS. Every new treatment and every new trial brings us closer to a world without this disease. The AMX0035 treatment will provide hope to countless people living with ALS and their families, but today there is still no cure.

Every 90 minutes someone is diagnosed with ALS while another dies from this disease. While we celebrate this victory today, tomorrow we will continue the search for a cure.

How ALS Patients Can Help

Once you’ve been diagnosed with ALS, it’s easy to feel like you just don’t have many options, but there are ways you can make a difference.

How You Can Help People With ALS

We all have a part to play in the fight against ALS! Your generous support funds critical research for better treatments and a cure for this disease. Here are a few ways you can help: