(Bloomberg) -- The world’s first therapy using gene-editing Crispr technology will be available for some patients in England through a deal between Vertex Pharmaceuticals Inc. and the government’s health system.
Casgevy, a one-time infusion developed by Vertex and Crispr Therapeutics AG, will be offered to patients with a serious form of beta-thalassemia, a hereditary blood disorder that requires repeated transfusions, according to the National Institute for Health and Care Excellence, the UK’s drug-cost regulator.
The gene-editing therapy makes precisely targeted changes in patients’ DNA, a months-long process that requires removing bone marrow and then a stem cell transplant. While the treatment holds the potential to cure certain blood disorders, NICE said it still isn’t convinced of its benefit or cost-effectiveness.
Patients will access the therapy under a five-year deal through the Innovative Medicines Fund, a program that gives England’s National Health Service a discount on Casgevy’s £1,651,000 ($2.1 million) list price. Around 460 patients with transfusion-dependent beta-thalassemia who are suitable for a stem cell transplant where donors aren’t available will be potentially eligible.
Research shows that beta-thalassemia patients who received Casgevy no longer needed transfusions, NICE said. But studies followed patients for a relatively short time and it isn’t clear how long its effects last.
The regulator has paused its assessment of Casgevy’s cost effectiveness in another blood disorder, sickle cell disease, while it assesses information Vertex submitted about related complications. NICE earlier declined to recommend the drug in a draft guidance.
The deal makes England only the fourth country where the therapy is available, following the US, Saudi Arabia and Bahrain. Access to innovative new drugs in England has become a point of contention, as the struggling NHS battles rising financial pressures.
Casgevy is one of the first few medicines to be offered to patients through the Innovative Medicines Fund, which was created to provide quicker access to potentially life-saving drugs. However, there has been little interest from industry as it requires drugmakers to keep providing drugs to patients even if the NHS decides the product won’t be recommended for continued use. This concern won’t apply to Casgevy, as it’s a one-time treaatment.
Pharma companies are pressuring the UK to reimburse certain expensive drugs. AstraZeneca Plc Chief Executive officer Pascal Soriot has warned that if the country doesn’t change the way it assesses new drugs, patients will continue to miss out and drugmakers might take their investments elsewhere.
(Updates with drug access details from seventh paragraph)
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