European Commission Approves First CRISPR Gene-Edited Therapy for Blood Disorder

The European Commission has approved CASGEVY™ (exagamglogene autotemcel) - the first CRISPR/Cas9 gene-edited therapy, for treating severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in patients aged 12 and older. This marks a significant regulatory milestone, offering over 8,000 patients in Europe a new treatment option. 

Developed by CRISPR Therapeutics and Vertex, CASGEVY is now advancing towards broader access and establishment of treatment centers across Europe.

The following article originally appeared in CRISPR Therapeutics.

CRISPR Therapeutics, a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

CASGEVY is the only genetic therapy approved for SCD and TDT patients in the European Union (EU) and with this approval, there are now more than 8,000 patients potentially eligible for treatment.

“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,” said Samarth Kulkarni, Chairman and Chief Executive Officer of CRISPR Therapeutics. “There is a significant burden of these diseases across Europe, and we look forward to bringing this therapy to these patients in need.”

Vertex leads global development, manufacturing, and commercialization of CASGEVY under the terms of a 60/40 profit sharing agreement with CRISPR Therapeutics. Vertex is working closely with national health authorities in the European Union (EU) to secure access for eligible patients as quickly as possible. Through this work, they have secured early access for eligible TDT patients in France ahead of the national reimbursement process. Vertex continues to engage with hospitals experienced in stem cell transplantation to establish a network of independently operated authorized treatment centers (ATCs) for the administration of CASGEVY. There are currently three activated ATCs in the EU with plans to activate a total of approximately 25 centers across Europe.

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