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.
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Gene Therapy: Bluebird’s Beti-Cel Sails Through US Panel Review On Strength of ‘Impressive’ Efficacy
Treatment is a potential game-changer for patients with transfusion-dependent beta-thalassemia, committee members said; panel was hesitant to extrapolate the hematological malignancies seen with bluebird’s other lentiviral vector programs to beti-cel.
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.
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.