One month ago on May 25, 2021, representatives from the FDA joined a We Can’t Wait Action Meeting. During this meeting, eight ALS advocates from across the US shared their personal experience with the disease to highlight why those with ALS need expedited access to experimental therapies.
Following the We Can’t Wait Action Meeting, the ALS Association sent a letter to the FDA that requests the agency to reaffirm their commitment to the 2019 ALS Guidance and provide details regarding how they have implemented the guidance.
The FDA’s Response to the Letter
Dr. Patrizia Cavazzoni, Director of the Center for Drug Evaluation and Research sent a response letter to the ALS Association on June 15, 2021. The letter acknowledges the “burden of ALS for patients, their families and caregivers,” noting there is an “unmet need for treatments” for the disease.
We can also assure you that we are exercising the regulatory flexibility described in FDA’s 2019 guidance for industry titled Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment (ALS Guidance) and we continue to commit to doing so,” Dr. Cavazzoni shared in the letter.
The FDA responded positively to the We Can’t Wait Action Meeting, but the ALS community still has grave concerns. The FDA has not clearly communicated what they are doing to expedite the approval of promising treatments for ALS patients. Yet they allow regulatory flexibility for other disease treatments.
The FDA recently used its regulatory flexibility to approve Aduhelm to treat Alzheimer’s. While this is great news for those living with Alzheimer’s and their loved ones, those living with ALS are left to wonder why the same urgency is not granted to them.
In the letter from the FDA, Dr. Cavazzoni shared that they are “committed to exercising [the guidance] when appropriate.” However, the agency does not appear to make any of those considerations for AMX0035. The drug met its primary and secondary endpoints in The Phase 2 Trial and is generally safe. Yet the FDA requires another clinical trial.
Health Canada is already working with Amylyx to bring the drug to market for Canadians with ALS in 2021. Yet Americans with ALS will have to wait years to access the drug. That is if the FDA even allows it to hit the market.
How We’re Moving Forward
The ALS Association is deeply committed to expediting the access to promising drug treatments to those living with ALS. We hope to achieve this by:
- Continuing to put public pressure on the FDA
- Work behind the scenes to expedite change
- Seek greater public accountability for the FDA
Read more about this response and how the ALS Association is implementing the above action items here. Our organization will continue to share important updates as they arise as we continue the fight to end ALS. We will not rest until we find a cure!
Read the Full Letter from the FDA
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.
