Bluebird Exits Europe, Casting Clouds Over Gene Therapy Commercial Effort
The company will prioritize US market for gene therapies; FDA also placed eli-cel for CALD on clinical hold following a case of MDS.
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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.
The cost-effectiveness watchdog now assumes an upfront $2.1m payment instead of five annual payments. The company switched gears to the US after the TDT gene therapy stumbled in Europe.