FDA reviewers on Ipsen's potential ultra-rare disease drug: Post-hoc analysis may be enough
Post-hoc analyses are generally a major no-no for the FDA, but agency reviewers signaled their openness to considering them in the case of Ipsen’s potential new therapy for patients with an ultra-rare genetic connective tissue disorder.
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee of outside experts will meet Wednesday to discuss whether it is reasonable to conclude that the drug, known as palovarotene, is safe and effective for patients with fibrodysplasia ossificans progressiva. The panelists will review one Phase II study with multiple open-label extension phases and one single-arm, open-label Phase III study with a natural history study as the external control to support the efficacy of palovarotene. Ipsen is requesting approval of 5 mg daily, with flare-up dosing at 20 mg daily for 4 weeks, followed by 10 mg daily for 8 weeks.
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