ALS Drugs: FDA Panel Weighed Less-Than-Ideal Data Against Amylyx’s Ability To Set Postapproval Price
National Institute on Aging’s Brian Traynor questioned whether Amylyx would seek to recoup its money on AMX0035 in 2-3 years, before the Phase III PHOENIX trial completes, or whether pricing would be based on long-term market expectations. NINDS’ Kenneth Fischbeck said Amylyx should reopen US sites in Phase III trial and broaden expanded access instead of seeking approval now.
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The US drug pricing watch dog issued a final assessment on Amylyx's AMX0035 and Mitsubishi Tanabe's Radicava for ALS.
Amidst bipartisan congressional pressure to approve ALS drugs, Billy Dunn, head of FDA’s neuroscience division, told advisory committee members that the law ‘demands’ flexibility in review of Amylyx’s AMX0035, and he came prepared with the citations.
In switching their votes from the first panel review in March, advisory committee members cited supportive additional analyses of a survival benefit, framing of the issues in the context of unmet need and regulatory flexibility, and the company’s statement that it would withdraw AMX0035 if the Phase III PHOENIX trial does not succeed.