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ALS Drugs: FDA Panel Weighed Less-Than-Ideal Data Against Amylyx’s Ability To Set Postapproval Price

 
• By Sue Sutter

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.

Pill container with money
Some advisory committee members worried about giving Amylyx free rein to price its ALS drug based on suboptimal data. • Source: Shutterstock

At the US Food and Drug Administration's second panel meeting on Amylyx Pharmaceuticals, Inc.’s amyotrophic lateral sclerosis drug AMX0035, some members of the Peripheral and Central Nervous System Drugs Advisory Committee sought to weigh a potential approval – and thus patient access – on the basis of less-than-ideal data against the company’s ability to charge for the product in a postapproval setting.

Although the FDA steadfastly maintains that cost is not a factor in its regulatory decision-making on the approvability of new drugs, it was top of mind for some committee members at the 7 September review of the ALS drug, who

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