FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednes­day ap­proved the first gene ther­a­py for a chron­ic con­di­tion — blue­bird bio’s new Zyn­te­glo (beti-cel) as a po­ten­tial­ly cu­ra­tive treat­ment for those with trans­fu­sion-de­pen­dent tha­lassemia.

The thumbs-up from the FDA fol­lows a unan­i­mous ad­comm vote in June, with out­side ex­perts point­ing to ex­tra­or­di­nary ef­fi­ca­cy, with 89% of sub­jects with TDT who re­ceived beti-cel hav­ing achieved trans­fu­sion in­de­pen­dence.

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