Daily Newsletter

14 February 2024

Daily Newsletter

14 February 2024

CRISPR gene therapy Casgevy secures approval in Europe

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

Robert Barrie February 13 2024

Vertex Pharmaceuticals and CRISPR Therapeutics have scored another regulatory approval for Casgevy (exagamglogene autotemcel) after the European Commission granted conditional marketing authorisation to the gene therapy.

The CRISPR treatment is the first gene therapy approved in Europe for sickle cell disease and transfusion-dependent beta-thalassemia (TDT).

The European Commission’s decision follows the positive opinion adopted by the European Medicine Agency (EMA) in December 2023. A condition marketing authorisation is valid for one year and can get renewed annually as more clinical data gets reported.

The UK Medicines and Healthcare products Regulatory Agency (MHRA) was the first authority to approve Casgevy in November 2023. The US Food and Drug Administration (FDA) followed suit with an initial approval for sickle cell disease in December 2023, and a TDT approval in January 2024.

In all three regions, the therapy is approved for the treatment of patients aged 12  and older with recurrent vaso-occlusive crises. TDT patients who can undergo haemopoietic stem cell transplantation but do not have a human leukocyte antigen-matched related donor are also eligible for the therapy. Vertex states that the latest approval makes available the treatment option to more than 8,000 patients.

Casgevy’s price has been under the spotlight since gaining regulatory approvals. Vertex and CRISPR have set a price of $2.2m for the one-time treatment. The companies have not made public the price in Europe, where access will be determined based on negotiations with national authorities.

However, Vertex stated early access for TDT patients in France has been organised ahead of national reimbursement. A total of 25 authorised treatment centres are slated to open in Europe, with three already operational.

While Vertex and CRISPR have the gene therapy market in sickle cell disease and TDT to themselves in Europe, the biotechs are competing with bluebird bio in the US. On the same day the FDA signed off on Casgevy in sickle cell disease, the agency also approved bluebird bio’s cell-based gene therapy Lyfgenia (lovotibeglogene autotemcel). The company already had its gene therapy Zynteglo approved to treat TDT in August 2022. Lyfgenia is priced at $3.1m while Zynteglo is priced at $2.8m.

Vertex’s CEO Reshma Kewalramani said: “Now our goal shifts to translating these approvals into real-world patient benefit and ensuring access and reimbursement across the globe.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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