We’re excited to share another milestone in the search for better drug treatments for ALS. Amylyx announced today that the company will soon submit a New Drug Application (NDA) for AMX0035 to the FDA. The ALS Association has been closely following the development of this treatment, even providing serious funding to propel its development.
Supporting the Development of a New Treatment
Many of these donations were received during the record-breaking ALS Ice Bucket Challenge in 2014. The ALS Association provided a grant of $750,000 for a clinical trial pilot in June 2016. The next month, a $1.46 million grant was provided to the Northeast ALS Consortium (NEALS) to fund the phase 2 CENTAUR clinical trial of AMX0035.
In September 2020, the concluded trial reports were released. They showed promise for this new drug. Those who received this AMX0035 experienced a significant slower progression of ALS than those who received the placebo. In terms of side effects, the drug presented itself as well-tolerated with a better benefit to risk balance. Read more about the CENTAUR phase 2 results here.
The Crucial Role of ALS Advocates
As soon as the results of phase 2 were released for AMX0035, ALS advocates jumped into action. In a team effort with I AM ALS, the ALS Association issued a petition. It urged the FDA and the drug company to make the treatment widely available as soon as possible. When the average lifespan is just 2 – 5 years after an ALS diagnosis, we can’t wait!
That’s just what ALS advocates communicated to the FDA just earlier this year in May. Eight ALS representatives from across the United States hosted a We Can’t Wait Action Meeting with the FDA. The meeting urged the FDA to act swiftly when it comes to approving new drug therapies for those living with ALS.
Our advocates play just as much as a role in the development and approval of new drugs as does the funding provided by generous supporters. With both these functions working in tandem, we can only hope for speedy approvals of future drug treatments.
What Comes Next?
Amylyx is currently conducting a third clinical trial (PHOENIX) of AMX0035; however, they can concurrently submit the NDA. It’s estimated that the drug company will submit this application sometime in the Fall 2021.
Following the application submission, the FDA will determine if they want to grant the new treatment approval. Or, contrarily, the FDA could require an additional study. This review process will take several months from the time of submission. In addition to the United States, Amylyx has also filed for approval of AMX0035 in Canada and the European Union.
In the meantime, the ALS Association plans to put pressure on the FDA to approve this treatment as soon as possible.
John Robinson from Parish, FL, a member of the board of trustees of the ALS Association and retired military veteran of nearly 32 years, was diagnosed with ALS in 2019. ALS not only affects the person with the disease but also their family caregivers. Both he and his wife can no longer serve their communities in the same way because of ALS. “The loss to society of their skills and humanity leaves holes never filled the same way again.”
Sunny Brous, a Texan with ALS, joined the call to share her perspective. Sunny was diagnosed with ALS in 2015 at the young age of 27. For Sunny, access to experimental treatments go beyond extending her own life. “Expanded access is how I can contribute to finding a cure for ALS. It takes my love for others and makes it a quantifiable action combining research and science and my dedication to the greatest good of people living with ALS.”
Sandy Morris, from Truckee, CA played a recorded message. She tearfully shared how many of her friends with ALS died waiting for access to life saving drug treatments. “Think about how much better it would be if we were able to die trying rather than die waiting,” she shared, urging the FDA to consider how their delays harm those living with ALS. “It’s not within my power to save my life—it’s within yours.”
Troy Fields from Champa Bay, FL was diagnosed with ALS in 2018. He noted how the considerations of benefits versus risk should be put in the hands of those living with ALS. “Because unless it is your own life on the line, you simply do not get it.”
Cari Meystrik from Nashville Tennessee was diagnosed with ALS in 1998 when she was only 28 years old. She has watched hundreds of friends die from this awful disease. “Would I jump to try a drug that might help me live longer of a disease that I am dying from if it has already proven to be sage and well tolerated but isn’t approved yet? Absolutely. Give me that chance.”
Jay Quinlan from New Orleans joined with his wife and caregiver Stephanie. “You have shown through your recent handling approvals for COVID vaccines that flexibility and expediting processes are possible.” He asked the FDA to use the same urgency when it comes to approving drug treatments for those living with ALS.
Tommy May from Pine Bluff, AR was diagnosed with ALS in 2005. Due to slow progressions, he was both unable to participate in clinical tries and frustrated with the lack of access to treatments. He discussed how those living with ALS are desperate for HOPE on their path. “We are not interested in the long-term. We are desperate for the here now.”
Larry Falivena of Apex, NC, has ALS caused by a mutation of the SOD1 gene. The fight for treatment access is not just for himself—it is also for his children. “I’m asking the FDA to help make sure my boys don’t have to worry about ALS.” He shares that even if the opportunity exists for just a small number of patients to have extended time on this earth that they need that chance.
