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!