ALS Association, I AM ALS Call on Amylyx, FDA to Make Promising New Drug Available for our ALS Community


The ALS Association and I AM ALS have launched a petition calling on the FDA and Amylyx Pharmaceuticals to bring AMX0035, a promising new drug developed by Amylyx, to market as soon as possible. Phase 2 testing of AMX0035 showed the drug slowed the progression of ALS. The petition asks that FDA agree to forego a phase 3 trial and instead, require rigorous follow-on testing as the drug is made broadly available to people living with the disease at the earliest possible opportunity. The results of its AMX0035 phase 2 trial were published on Wednesday in the New England Journal of Medicine.

AMX0035 is an oral therapy dissolved in water that combines two existing drugs, sodium phenylbutyrate and taurursodiol. Trial results indicate the drug cocktail slowed ALS progression in a statistically meaningful and clinically relevant way and that the drug combination is generally safe and well-tolerated. Moreover, the safety of the underlying drugs that make up AMX0035 has been well established clinically.  


The phase 2 trial included 137 people with ALS across 25 medical centers that are part of the Northeast ALS (NEALS) continuum. People with ALS who received AMX0035 performed 2 points better on the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), which could mean the difference between a person with ALS being able to feed themselves versus being fed, or the difference between needing a wheelchair versus not needing one.  

Given the large phase 2 sample size, clinically relevant slowing of disease progression, and significant evidence that the drug is safe, the Association and I AM ALS launched the petition asking that Amylyx and the FDA move with urgency make this drug available for all people living with ALS. People with ALS cannot wait for a phase 3 trial to conclude given the promising results shows in phase 2. Since the FDA has emphasized the appropriateness of exercising flexibility when dealing with serious diseases and unmet medical needs, there is no reason to withhold this promising drug from the ALS community. Read and sign the petition:

The ALS Association supported the development of AMX0035 with a $2.2 million grant in 2016 – one of the first research grants funded with ALS Ice Bucket Challenge donations. As a standard provision in philanthropic support for drug development, the grant included a repayment provision allowing the Association to recover up to 150 percent of its support, or up to $3.3 million.  The funds will be reinvested into ongoing global research into treatments and, ultimately, a cure.  


Submitted by: Christina C. on Wed, 12/30/2020

I will do anything I can to help this cause

Submitted by: Vicki A. on Tue, 01/26/2021

So excited on ANY news for a cure! Thank you for your work!

Submitted by: FRANCES C. on Sat, 03/27/2021

Why is this drug not available for use at the present?

Submitted by: Amy L. on Tue, 03/30/2021

Hi Frances. AMX0035 has not been approved by the FDA. The ALS Association has asked the FDA to address this issue with urgency, by approving the drug prior to a phase 3 trial.

Submitted by: Janette W. on Tue, 03/29/2022

Hello. My 54 year old cousin was just diagnosed with ALS. We are all shocked and so saddened by this news. She lives in another state. Can you please give us some advice about what we should or should not say or do and how we can help her in a meaningful way.

Submitted by: Amy L. on Wed, 03/30/2022

Hi Janette. So sorry to hear that your cousin has been diagnosed with ALS. You can learn more about ALS here:

Your cousin's local ALS Association chapter can provide your family with information, resources and support. Find their contact information at

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