FDA Panel Approves Ultra-Rare Bone Disease Therapy Despite Data Concerns

An FDA advisory committee voted 10 to 4 to recommend Ipsen Biopharmaceuticals’ palovarotene as an effective therapy for the ultra-rare bone disease fibrodysplasia ossificans progressiva (FOP), which causes tendons and ligaments to be replaced by bone.

Before Wednesday’s meeting, members of the Endocrinologic and Metabolic Drugs Advisory Committee remarked in briefing documents that Ipsen’s late-stage study cohort contained an “unexpected” increase in disease flare-ups, which are “frequently triggered by local inflammation or trauma and are characterized by ossification of muscle and other soft tissues.” That flare-ups preceding bone formation in patients given the drug is a worrying correlation, the committee said.

Multiple panelists remarked that although there are no currently approved targeted therapies for treating FOP and there exists a dire patient need for treatments, they were troubled by Ipsen’s pivotal late-stage study data.

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