Ipsen's rare disease candidate rejected after mounting setbacks, delays

Ipsen’s “largely derisked” rare disease candidate was evidently still too risky for regulators, with the FDA rejecting the company’s new drug application for palovarotene.

The decision by regulators, announced by the company late Friday night, comes two months after the FDA delayed an advisory panel meeting to discuss palovarotene and the agency's request for additional data. Ipsen said the complete response letter is related to that previous request, and it intends to respond by the first quarter of 2023, triggering a new six-month review cycle. The company also said the FDA has not yet rescheduled the advisory meeting.