NICE Approves Vertex, CRISPR's Casgevy for Beta Thalassemia, Not Sickle Cell

On the challenging path to making the gene therapy Casgevy accessible in the U.K., Vertex and CRISPR Therapeutics have achieved a significant milestone. England’s National Institute for Health and Care Excellence (NICE) has given a favorable recommendation for one of Casgevy's approved uses. In its final draft guidance, NICE endorsed Casgevy, also known as exa-cel, as a cost-effective, one-time treatment for transfusion-dependent beta thalassemia. However, in March, the agency did not approve the therapy for its sickle cell disease indication, stating it needed to gather more data on the treatment’s effectiveness.

NICE has now approved Casgevy for beta thalassemia patients aged 12 and older who are suitable candidates for a hematopoietic stem cell transplant but lack a matching donor. The therapy will be funded through England’s Innovative Medicines Fund (IMF), which supports non-cancer drugs while additional data on their efficacy is collected. As a result, Casgevy will be immediately available through the IMF for up to 460 eligible patients, according to NICE. The drug is priced at 1,651,000 pounds sterling (approximately $2 million), but a confidential discount will be applied.

Despite some uncertainties about the long-term benefits of the therapy, NICE's committee concluded that Casgevy could potentially cure some individuals with transfusion-dependent beta thalassemia, alleviating them from the continuous need for blood transfusions. Helen Knight, NICE’s director of medicines evaluation, emphasized this potential in a public statement.

Casgevy holds the distinction of being the world’s first CRISPR-based gene therapy. It received U.K. authorization for its genetic blood disorder indications last November. Given the high list price and NICE's previous decisions not to endorse Vertex’s cystic fibrosis drugs, the company faced significant challenges in access negotiations.

In the United States, Casgevy was approved in December following an advisory committee vote by the FDA in October. To date, about 20 patients have initiated their treatment with the initial cell collection process. Vertex has established over 35 active treatment centers globally. Approximately 35,000 patients in the U.S. and Europe have been identified as eligible for Casgevy.