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.
Bluebird’s Eli-Cel Gene Therapy Gets US Panel Nod For Some Cerebral Adrenoleukodystrophy Patients
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.
Bluebird’s Beta-Thalassemia Gene Therapy Faces US Panel Scrutiny On Hematologic Malignancy Risk
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.