FDA must act urgently on new therapy approval.

Amyotrophic lateral sclerosis (ALS) is a horrific neurodegenerative disease that affects over 30,000 Americans. It kills so swiftly that people with ALS typically live for just two to five years after diagnosis.

Our organizations are on an urgent mission to change ALS from universal death sentence to a livable disease until cures are found. The FDA is currently reviewing a drug – Amylyx Pharmaceuticals Inc.’s AMX0035 – that could help bring that goal one step closer to reality. The case for approval in the United States – especially given the recent approval of AMX0035 by Health Canada – is clear, and the time is now. We urge the FDA to act swiftly to complete its review in advance of a recently extended decision deadline and approve AMX0035.     

AMX0035 is a combination of two previously approved drugs, now formulated together for more powerful effect. In a large, placebo-controlled study conducted at 25 of the top ALS clinics in the world, AMX0035 was found to be safe and effective at slowing loss of function with minimal side effects – and study participants on the drug lived longer by 10 to 18 months.

For a disease with a median survival time from diagnosis of two to five years and no known cure, the potential for up to 1.5 years more life is priceless. It is additional time for those suffering with ALS to spend with their families and loved ones. As one point of comparison, a 2020 JAMA review found that among 92 cancer drugs approved between 2000 and 2016, the average survival benefit was less than 3 months.

AMX0035 is effective and safe for people living with ALS. AMX0035 was evaluated in a placebo-controlled study of 137 patients conducted by expert ALS clinicians at 25 of the top ALS clinics in the world. First, it met its primary endpoint – an endpoint specifically highlighted for use in the FDA’s own drug development guidance document. Second, more promising results continue to emerge, such as reduced risk of first hospitalization. Third, while the FDA posed questions about the data at a public Advisory Committee meeting these questions have been answered in support of approval, such as in this Annals of Neurology editorial by two leading ALS clinician-scientists, Dr. Merit Cudkowicz and Dr. Jeremy Shefner. Finally, dozens more clinicians around the country have signed a letter stating that they want this drug approved so they can prescribe it to their patients.

The need is great. ALS will strike roughly one in 400 people in their lifetimes, and as of today brings a universal death sentence with a median time to death from diagnosis of two to five years. Sometimes, it is as short as months. Without fail, ALS robs people of their capacity to use their arms, their legs, to speak, to eat, and – ultimately – to breathe.  A drug that slows decline and extends life means more time to hug a grandchild, more time to attend a loved one’s wedding, or the dignity of being able to feed oneself.

The FDA has tools to act with urgency and flexibility for ALS. In 2019, the FDA released ALS guidance that notes the “FDA has long stressed the appropriateness of exercising regulatory flexibility in applying the statutory standards to drugs for serious diseases with unmet medical needs, while preserving appropriate assurance of safety and effectiveness.” In simple terms: the FDA can act swiftly to approve new treatments for ALS. It goes on specifically to highlight the outcome measurement tool used in the AMX0035 trial as one for priority use. Additionally, a confirmatory study that will provide more data is underway and will continue after AMX0035 is approved. It will, however, take several years to complete. Waiting years longer for its results would deny 30,000+ Americans living with ALS today access to a drug that may extend their lives by 10+ months. That is the equivalent of closing the door on more than 24,000 years of additional life.

While AMX0035 is not a cure, its effects could be transformative for people living with ALS. These results are the best the ALS scientists have seen in years, the need is great, and the FDA has the authority to act with urgency and flexibility to combat ALS. The FDA must use that authority to give people with ALS a chance for a longer life.

Answer ALS Foundation
The ALS Association
I AM ALS
Les Turner ALS Foundation
Team Gleason