US FDA Doesn’t Need Conditional Approval Pathway, Cavazzoni Tells House Committee
CDER director says agency has many tools to expedite development and does not endorse adding a pathway similar to EU’s conditional approval. Neurodegenerative diseases hearing in Energy & Commerce Committee focuses more on ALS than Aduhelm.
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Promising Pathway Act Would Fill Gap For Rare Disease Treatments That Don’t Fit Accelerated Approval, Advocates Say
Bill would enable two-year provisional approval of drugs to treat life-threatening illnesses, such as ALS, based on substantial evidence of safety and early evidence of efficacy. At a Senate committee hearing, rare disease advocates said it would remedy the FDA’s inconsistent application of regulatory flexibility, but a bioethicist cautions it would lower the bar for approval.
A bipartisan letter from several key legislators is pushing the US FDA to match its actions to its rhetoric when it comes to the handling of ALS drug reviews. Amylyx’ pending AMX0035 isn’t mentioned but seems unavoidably the subject.
The challenges to reforming the US FDA Accelerated Approval pathway are nicely illustrated by comparing the first two legislative proposals offered by the Democratic and Republican leadership. The bills are almost exactly opposite in intent – though perhaps there is some room for consensus on first steps.