Accelerated Approval For Bluebird’s Skysona Gives Teeth To US FDA Data Questions
Two required confirmatory studies will bolster application that relied on historical controls for orphan neurodegenerative disease.
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RWE On The Runway: Trio Of Upcoming Approval Decisions Will Test US FDA’s Thinking On RWE For Efficacy
Real-world evidence provides external controls for bluebird bio’s eli-cel and Ipsen’s palovarotene, while Reata used RWE to address FDA concerns about the pivotal single-arm study of omaveloxolone. In the post-market, Intercept is gearing up to seek full approval of Ocaliva relying in part on claims database.
Committee endorses use for those who lack a matched donor for allogeneic hematopoietic stem cell transplant and says the treatment should be an option for patients with a matched, but unrelated, donor.
Biogen/Eisai’s Phase III data is on track for a Q1 2023 submission, potentially just weeks after lecanemab is expected to gain accelerated approval for early Alzheimer’s.