Vertex Pharmaceuticals and NHS England have struck a “landmark” agreement to provide gene therapy Casgevy to beta thalassemia patients, starting with access for up to 460 people.
The deal comes as the UK’s National Institute for Health and Care Excellence’s final draft guidance on the subject released Thursday recommends Casgevy for people aged 12 and older with severe beta thalassemia who need regular blood transfusions and have no donor available for a transplant.
This is a notable win for Vertex because, in March, NICE published a separate draft guidance saying it does not yet recommend Casgevy for sickle cell disease due to lingering questions about its durability. NICE is responsible for weighing the value of money against a drug’s clinical benefit.
Casgevy has a list price of $2.2 million in the US but will be sold at an undisclosed discount to the NHS under the recent deal. The medicine uses CRISPR technology to remove and edit the faulty gene that causes patients’ bodies to produce less hemoglobin.
Last week, Vertex said that around 20 patients have had their cells collected so far for Casgevy treatment. On the back of the new UK deal, the rollout will “accelerate” to reach up to 460 people.
In a welcome surprise for Vertex and partner CRISPR Therapeutics, the UK’s MHRA was the first regulatory body anywhere in the world — even ahead of the FDA by several weeks — to approve Casgevy in November last year. In the UK and US, it is approved for sickle cell disease and beta thalassemia.
Ludovic Fenaux“Through collaboration with NHS England and NICE, we have reached this landmark agreement that recognizes the value a one-time treatment can provide to patients, their families and the health care system,” Vertex’s SVP Ludovic Fenaux said in a company release.
According to NHS England, there are around 2,300 people with thalassemia in the UK, including 800 with severe disease who depend on regular blood transfusions. The largest ethnic groups affected in the UK are people from an Indian, Pakistani or Bangladeshi background.
Severe forms of beta thalassemia can cause delayed growth, problems with bones and the endocrine system and shorten the length of a patient’s life.
Elsewhere, bluebird bio’s gene therapy for sickle cell disease, Lyfgenia, secured FDA approval in December at the same time as Casgevy. The drug has not yet been approved in the UK.
Editor’s note: The headline was updated to correct that the deal was between Vertex and the NHS, and not with NICE.