BLUE - Bluebird bio spikes on FDA briefing docs for gene therapy candidate

Update: Bluebird bio (NASDAQ:BLUE) shares gained after the FDA reviewers issued favorable remarks on the company’s gene therapy candidate for ?-thalassemia, beti-cel. Commenting on its efficacy, the FDA staff wrote that the data in its marketing application “support the effectiveness” of the drug for the targeted indication with a “clinically meaningful benefit” in terms of transfusion independence in late-stage trials. However, the FDA raised concerns over the company’s other gene therapy candidate, eli-cel for cerebral adrenoleukodystrophy (CALD). CALD is a rare neurodegenerative X-linked metabolic disease for which there are no approved treatments currently. “The overall benefit-risk profile of eli-cel is difficult to characterize because of the uncertain benefit and the uncertain magnitude of the risk of MDS,” the reviewers wrote, referring to a fatal malignancy detected in the trials that supported its marketing application. Previously: Bluebird bio (BLUE), a commercial-stage biotech focused on genetic diseases, announced on Tuesday that the FDA posted

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Bluebird bio spikes on FDA briefing docs for gene therapy candidate