Bluebird bio may finally get a gene therapy approved in the US

Bluebird bio may, at long last, get a gene therapy approved in the US.

The Cambridge, MA biotech announced Monday that the FDA had accepted and given priority review for Zynteglo, its gene therapy for the rare blood disorder beta-thalassemia. The announcement sets up an expedited FDA decision by May 20, 2022.

A priority review doesn’t guarantee approval, and more than a few recent biotechs have been spurned after receiving the designation, including Incyte, Sesen, and Provention. But it represents a substantial step forward for a therapy that has seen repeated setbacks and an application that was slapped down with a refuse-to-file letter.

The approval rate for candidates given FDA priority review hovered between around 70% and 90% between 2011 and 2019, compared to a little over 60% for drugs given the 10-month standard reviews.

Nick Leschly

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