Ipsen’s Palovarotene Ultra-Rare Bone Disorder Drug Faces Hurdles At US FDA Adcomm

 
• By Brenda Sandburg

Agency questions whether post hoc analyses and use of natural history subjects as an external control group in a Phase III study support effectiveness of palovarotene; cites apparent increased incidence of flare-up events as a key safety issue.

Boat uncertainty
Fate of Ipsen's rare disease drug palovarotene is uncertain given questions raised about study data • Source: Shutterstock

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