FDA’s about-face on Stealth a positive sign for rare disease therapies?

Following FDA’s denial of a request to reconsider a complete response letter, the agency is now promising an unusually quick review for Stealth’s rare disease therapy, after urgent messaging from the company and pressure from patient advocates to address an unmet need.

Although FDA has been signaling that it intends to employ flexible standards for rare disease therapies, patient advocates have expressed concerns about what they see as mixed messaging from agency leadership, in particular related to the transparency and predictability in regulatory standards...