The US Food and Drug Administration approvals of Vertex Pharmaceuticals Incorporated/CCRISPR Therapeutics AG’s gene editing medicine Casgevy (exagamglogene-autotemcel, exa-cel) and bluebird bio inc.’s gene therapy Lyfgenia (lovotibeglogene autotemcel, lovo-cel) gave doctors two new options to consider for patients with sickle cell disease (SCD) and a lot to discuss about how these novel treatments will be used going forward during the recent American Society of Hematology (ASH) annual meeting.
Key Takeaways
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Vertex/CRISPR’s Casgevy and bluebird’s Lyfgenia are the first two genetic medicines to treat sickle cell disease, giving doctors and patients a new potentially curative option, but at high costs and with complex administration that may limit the treatments’ use, both within and outside the US.
The 8 December approvals for both products to treat SCD patients aged 12 and older with recurrent vaso-occlusive crises (VOCs)...
