FDA to Decide on Bluebird Bio’s Gene Therapy for Sickle Cell Disease

Bluebird Bio’s gene therapy for Sickle Cell Disease (SCD), lovotibeglogene autotemcel (lovo-cel), is awaiting FDA approval, with the decision expected by December 20. The FDA has opted not to hold an advisory committee meeting before the decision. Lovo-cel, backed by efficacy results from 36 patients and safety data from 50 patients, has the most extended follow-up of any gene therapy program for SCD.

In 2021 and earlier, regulatory requests and a partial clinical hold delayed the therapy. If approved, lovo-cel would become Bluebird’s third ex-vivo gene therapy, following Zynteglo and Skysona. However, it could face competition from Vertex and CRISPR Therapeutics’ exa-cel, awaiting the FDA decision on December 8. Bluebird has warned of its survival struggles but expects its cash runway to extend until late 2024.

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