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Complete Response Letters See Uptick With US FDA's Thumbs Down For Waylivra

 
• By Michael Cipriano

In first half of 2018, CRLs were rarely issued for CDER-regulated novel agents, but FDA's rejection of Waylivra marks second in two weeks.

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While novel product rebuffs by the US FDA's Center for Drug Evaluation and Research (CDER) have been rare occurrences for most of 2018, a complete response letter (CRL) for Akcea Therapeutics Inc.'s Waylivra (volanesorsen) was the second for a new molecular entity (NME) in two weeks, following that for Mallinckrodt PLC's stannsoporfin.

Waylivra, designed for the treatment of familial chylomicronemia syndrome (FCS), previously escaped a US FDA advisory committee meeting with a 12-8 vote in favor of approval. (Also see "Akcea Waylivra's Surrogate Endpoint Gains Support At US FDA Advisory Committee " - Pink Sheet, 11 May, 2018.) However, the agency struck a more skeptical tone in its briefing documents, where it raised questions about triglyceride levels as a surrogate endpoint, the adequacy of the pivotal trial and a thrombocytopenia safety signal

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