The ALS Association and I AM ALS on Friday submitted a petition to the Food and Drug Administration calling on the agency and Amylyx Pharmaceuticals to act swiftly and with urgency to make AMX0035 available as soon as possible. The petition was signed by more than 50,000 people from across the country who have been affected by ALS.
Phase 2 clinical trials of AMX0035 have shown the treatment to be safe and effective at slowing down disease progression, and a follow-up open label extension study showed a statistically significant 6.5 month increase in survivability.
“I agree with and understand the FDA’s interest in protecting people from harmful or misleading drugs. ALS should be treated differently, however, and the FDA has acknowledged that. People living with ALS are willing to tolerate much greater risk and have expressed this to the FDA for years. I can attest to that. We need the FDA and Amylyx to work together to make AMX0035 available to people with ALS as soon as possible,” said Tommy May, a member of The ALS Association’s national board of trustees. May was diagnosed with ALS in 2005.
Noting that people with ALS are willing to accept more risk until effective treatments are found and that AMX0035 meets endpoints that are important to people living with ALS – including clinically meaningful delay in ALS progress, a strong safety profile, significant survival benefit, and an advance beyond or on top of currently available ALS care – the petition urges FDA and Amylyx to exercise every possible option to speed approval of this promising treatment to the market.
“The ALS community has united to ask the FDA and Amylyx to make AMX0035 available as soon as possible. Given the promise that AMX0035 shows in slowing progression and its safety record, the community should not have to wait several more years for another clinical trial. Thousands of us who could have been helped will have passed from this devastating disease,” said Larry Falivena, a member of The ALS Association’s national Board of Trustees.