Vertex/CRISPR Nab First-Ever Gene Editing FDA Nod, Overshadow Bluebird’s Same-Day Win

The two first-ever therapies to offer sickle cell disease patients the possibility of lifetime relief from painful vaso-occlusive events or crises are hitting the market, but face tough commercial challenges.

3d rendered medical illustration of a sickle cell — Both Casgevy and Lyfgenia are approved for patients aged 12 and up • Source: Shutterstock

Two sickle cell disease therapies won US Food and Drug Administration approval on 8 December but bluebird bio inc.’s gene therapy Lyfgenia (lovotibeglogene autotemcel, lovo-cel) stumbled out of the gate with higher pricing, a black box warning and no priority review voucher, while Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG may have a relatively easier path with Casgevy (exagamglogene-autotemcel, exa-cel), the first-ever CRISPR gene-edited medicine approved in the US.

Key Takeaways

Vertex/CRISPR’s Casgevy and bluebird’s Lyfgenia were approved in the US to treat sickle cell disease, the first potentially curative treatments for SCD.

Both therapies’ approvals – for SCD patients aged 12 and older with recurrent vaso-occlusive crises (VOCs) or a history of...