Melphalan flufenamide
A myeloma drug given accelerated approval in 2021 and withdrawn in the US months later when its confirmatory trial suggested it shortened survival.
Melflufen is a lipophilic peptide-conjugated alkylator activated by aminopeptidases enriched in myeloma cells. Accelerated approval (February 2021) rested on the single-arm HORIZON response rate. The confirmatory OCEAN trial met its PFS endpoint but showed an overall survival detriment in a subgroup (patients with prior autologous transplant), leading to a partial clinical hold, an ODAC vote against, and US withdrawal (2021; formal FDA withdrawal 2024). It remains approved in the EU with restrictions.
Lesson: accelerated approval on response rate carries real risk when the confirmatory trial shows harm; the same subgroup could be a true signal or noise, and regulators in the US and EU read it differently.
Trials
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Latest papers
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