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Accelerated Approval: ALS Drug Could Test New Congressional Directive On Survival

 
• By Sarah Karlin-Smith

Clene leadership spoke with the Pink Sheet about its push for accelerated approval for its ALS nanoparticle treatment. A new nudge from Congress asking the US FDA to consider survival data in accelerated approval could help.

close up of car speedometer at 160 km/h
House appropriators recently directed FDA to consider survivability data for accelerated approvals. • Source: Shutterstock

The US Food and Drug Administration is under new pressure from Congress to consider “survivability” as an endpoint that could be used for accelerated approval, and Clene, Inc.’s ALS drug could quickly turn that pressure from abstract to concrete reality.

Key Takeaways

  • Congress is putting new pressure on FDA to use accelerated approval for neurological diseases with a focus on using survival data for these approvals in ALS.

  • The House Appropriation Committee’s interpretation of the accelerated approval statute seems to veer from past understanding of the law and FDA’s long-held interpretation

The House Appropriations Committee directed FDA to “clarify its guidance that when considering new therapies for accelerated approval, including in ALS, survivability is a clinical endpoint that will be considered consistent with the current statute,” in a 10 July appropriations report

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