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The European Commission has granted conditional marketing authorization for CASGEVY. CASGEVY will be the only genetic therapy approved for sickle cell disease and beta-thalassemia patients in the European Union.
CRISPR Therapeutics has announced the European Commission’s conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]). CASGEVY is greenlit for patients aged 12 and above with severe sickle cell disease (SCD) or transfusion-dependent beta-thalassemia (TDT), expanding treatment options where hematopoietic stem cell (HSC) transplantation isn’t feasible due to the unavailability of a matched donor.
This approval opens treatment options for over 8,000 eligible patients in the European Union (EU), aged 12 years and older with severe SCD or TDT.
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“The approval by the European Commission is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for patients with severe sickle cell disease and transfusion-dependent beta-thalassemia,” commented Samarth Kulkarni, Chairman and CEO of CRISPR Therapeutics.
Vertex Pharmaceuticals, in collaboration with CRISPR Therapeutics, oversees the global development, manufacturing, and commercialization of CASGEVY, aiming to secure rapid access for eligible patients across EU nations. Vertex has already secured early access for eligible TDT patients in France and is working to establish a network of authorized treatment centers (ATCs) across Europe, with three centers already activated and plans for approximately 25 more across Europe.
SCD is a progressive genetic disease that causes severe pain, organ damage, and shortened lifespan due to abnormal red blood cells. Vaso-occlusive crises (VOCs) are a hallmark of SCD, leading to debilitating pain and organ complications. Stem cell transplant offers a curative option, but its availability is limited by the scarcity of matched donors. The approval of CASGEVY represents a significant advancement in addressing the unmet medical needs of SCD and TDT patients in Europe.
“There is a significant burden of these diseases across Europe, and we look forward to bringing this therapy to these patients in need,” stated Kulkarni.
Source: CRISPR Therapeutics Press Release
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