On the rocky road to ensuring access to gene therapy Casgevy in the U.K., Vertex and CRISPR Therapeutics are making inroads by securing a thumbs-up from England’s National Institute for Health and Care Excellence (NICE) for one of the treatment's two approved indications.
In final draft guidance, NICE opted to recommend Casgevy, which is also known as exa-cel, as a cost-efficient, one-time treatment for transfusion-dependent beta thalassemia.
In March, the agency declined to endorse the therapy in its sickle cell disease indication. At the time, the regulator said it was “exploring further data collection” on the therapy’s effectiveness.
Now, NICE has signed off on Casgevy for patients 12 years and older with beta thalassemia for whom a hematopoietic stem cell transplant (or a blood and bone marrow transplant) is appropriate but no donor is available.
The therapy will be available through England’s Innovative Medicines Fund (IMF), which pays for non-cancer drugs while collecting further data on the treatments.
That means Casgevy will be covered “immediately” by the IMF to speed up the rollout for up to 460 people eligible for the treatment, according to NICE. The drug carries a list price of 1,651,000 pounds sterling ($2 million), which will be subject to a confidential discount.
Despite “some uncertainties” with the evidence supporting the therapy’s long-term benefits, NICE's committee determined that Casgevy “could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions,” NICE’s director of medicines evaluation, Helen Knight, explained in a release.
Casgevy is the world’s first CRISPR-based gene therapy. The drug scored its U.K. authorization for both of its genetic blood disorder indications last November. With a high list price and NICE's prior history of passing on Vertex's cystic fibrosis drugs, the company had its work cut out in the access negotiations.
Meanwhile, in the U.S., Casgevy was approved in December following an October FDA advisory committee vote. So far, about 20 patients have begun their treatment journey with the cell collection process, and Vertex has set up more than 35 active treatment centers across the globe. About 35,000 patients have been identified as being eligible for the drug in the U.S. and Europe alone.