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Accelerated Approval For Bluebird’s Skysona Gives Teeth To US FDA Data Questions

Executive Summary

Two required confirmatory studies will bolster application that relied on historical controls for orphan neurodegenerative disease.

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Bluebird’s Lost Voucher: Lyfgenia Contains Same ‘Active Ingredient’ As Zynteglo, US FDA Says

Surprise denial of rare pediatric disease priority review voucher could prompt calls for the agency to re-examine how it interprets the term ‘active ingredient’ for gene therapies. Bluebird asserts there are significant differences between its gene therapies for treating sickle cell and beta-thalassemia.

CAR-T Malignancy Reports Could Temper Excitement For Use Beyond Oncology

US FDA has received a total of 19 reports of T-cell malignancy involving BCMA- or CD19-directed autologous CAR-T cell immunotherapies and is evaluating the need for regulatory action. Focus on lentiviral and retroviral vectors could extend to safety of other types of gene therapies.

Gene Therapies: US FDA Has Used Accelerated Approval Four Times Based On Intermediate Clinical Endpoints

Three of the accelerated approvals were for CAR-T products, a class that the agency considers to be gene therapies. The FDA’s September approval of bluebird bio’s Skysona for cerebral adrenoleukodystrophy was the lone non-cancer product in the group. Pink Sheet infographic tracks the required confirmatory studies and timelines for all four products.

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