Gene Therapy: Bluebird’s Eli-Cel Efficacy Uncertain, Malignancy Risk Concerning, US FDA Says
Interpretation of efficacy from single-arm studies in patients with cerebral adrenoleukodsytrophy complicated by comparability with external controls and limited follow-up, FDA says in advisory committee briefing documents which also cite three cases of myelodysplastic syndrome and raise broader questions about lentiviral vector safety.
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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.
Although there were no cases of hematological malignancy reported and no evidence of insertional oncogenesis in patients treated with beti-cel, FDA raises a theoretical risk due to delayed platelet engraftment, as well as malignancies seen in other lentiviral vector programs.