Vertex & CRISPR’s Casgevy Given the Green Light by NICE

Vertex and CRISPR Therapeutics are making headway on the difficult path to assuring the availability of gene therapy Casgevy in the U.K. They have received a thumbs-up from England’s National Institute for Health and Care Excellence (NICE) for one of the therapy’s two authorized indications.

Casgevy, which is also known as exa-cel, was selected by NICE as a cost-effective, one-time therapy option for transfusion-dependent beta thalassemia in the final draft of recommendations.

The agency decided not to provide its approval to the medicine for the sickle cell disease indication in March. When asked about the efficacy of the therapy at the time, the regulatory body stated that it was looking into more extensive data collection.

Casgevy has now been approved by the NICE for use in patients aged 12 years and older who have beta thalassemia and would benefit from a hematopoietic stem cell transplantation, but there’s an unavailability of any donor.

There will be access to the treatment through England’s Innovative Medicines Fund (IMF), which provides funding for non-cancer medications while gathering additional information on the therapies.

According to NICE, this indicates that the International Monetary Fund will cover Casgevy “immediately” in order to expedite the deployment of the therapy for up to 460 individuals who are qualified for it. The drug has an advertised price of 1,651,000 pounds sterling, equivalent to $2 million.

NICE’s committee came to the conclusion that Casgevy “could represent a potential cure for some people with transfusion-dependent beta-thalassemia, freeing them from the burden and risks of needing regular blood transfusions,” as stated by Helen Knight, the director of medicines assessment at the firm, in a press release. This was despite the fact that there were some uncertainties regarding the proof that supported the long-term advantages of the therapy.

Casgevy is the first gene treatment based on CRISPR. In November last year, the medicine was granted permission in the U.K. for both of its indications with regard to hereditary blood disorders. Due to the high list price and the fact that NICE had previously rejected Vertex’s cystic fibrosis medications, the company had its work cut out in order to gain access to the market.

During this time, Casgevy was granted approval in the U.S. in December, after a vote placed by the FDA advisory committee in October. Up to this point, about 20 patients have started their treatment path with the cell collection procedure, and Vertex has established more than 35 treatment facilities that are currently operational all around the world. In the U.S. and Europe alone, around 35,000 individuals have been confirmed to be suitable for the therapy.

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