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CRISPR gene therapy Casgevy granted European approval 

CRISPR gene therapy Casgevy granted European approval 

USA – Vertex Pharmaceuticals and CRISPR Therapeutics have achieved another milestone with the European Commission granting conditional marketing authorization to Casgevy (exagamglogene autotemcel). 

The approval makes Casgevy the only gene therapy available in Europe for sickle cell disease and transfusion-dependent beta-thalassemia (TDT). 

 The decision by the European Commission closely follows the positive opinion endorsed by the European Medicine Agency (EMA) in December 2023.  

Notably, the conditional marketing authorization, valid for one year, opens doors for annual renewals as more clinical data becomes available. 

The UK Medicines and Healthcare products Regulatory Agency (MHRA) had already paved the way by granting approval to Casgevy in November 2023.  

Similarly, the US Food and Drug Administration (FDA) swiftly followed suit with approvals for sickle cell disease in December 2023 and TDT in January 2024. 

Across all three regions, Casgevy is sanctioned for treating patients aged 12 and older experiencing recurrent vaso-occlusive crises. 

 Additionally, TDT patients, eligible for haemopoietic stem cell transplantation but lacking a matched related donor, can benefit from this therapy. 

Pricing concerns surface 

Vertex Pharmaceuticals has emphasized that this latest approval will significantly broaden treatment options, potentially benefiting over 8,000 patients.  

However, pricing concerns have surfaced since the therapy’s approval. 

 Vertex and CRISPR have set the price at $2.2 million for this one-time treatment, though the specific price in Europe remains undisclosed pending negotiations with national authorities. 

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Despite pricing uncertainties, early access initiatives for TDT patients in France have been established ahead of national reimbursement, with plans for a network of authorized treatment centers across Europe.  

Currently, three centers are operational, with 25 anticipated to be fully operational soon. 

In the competitive landscape, Vertex and CRISPR face competition primarily from bluebird bio in the US market.  

Coinciding with Casgevy’s FDA approval for sickle cell disease, the FDA also greenlit bluebird bio’s Lyfgenia (lovotibeglogene autotemcel), adding to their existing therapy Zynteglo approved for TDT treatment in August 2022. While Lyfgenia is priced at $3.1 million, Zynteglo carries a price tag of $2.8 million. 

Reshma Kewalramani, CEO of Vertex, looks beyond approval while saying that his focus was on translating these milestones into tangible patient benefits globally to ensure widespread access and reimbursement. 

 “Now our goal shifts to translating these approvals into real-world patient benefit and ensuring access and reimbursement across the globe.”

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