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
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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.
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Casgevy, the first-ever FDA-approved gene-editing medicine, may have a pricing advantage with a $900,000 lower list price than Lyfgenia’s $3.1m cost; the Vertex product also does not have a black box warning and will be marketed globally, not just in the US like bluebird’s gene therapy
Both therapies’ approvals – for SCD patients aged 12 and older with recurrent vaso-occlusive crises (VOCs) or a history of vaso-occlusive events (VOEs) – were greeted with enthusiasm, but investors and biopharmaceutical industry observers were stunned by
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