Following positive guidance issued by the National Institute for Health and Care Excellence (NICE), the CRISPR-based gene therapy Casgevy has been made available to patients with transfusion-dependent beta thalassemia (TDT) in England.
Today (8 August), NICE released their final draft guidance recommending Casgevy coverage via the NHS. Casgevy was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The recommendation concerns patients aged 12 and older with severe beta-thalassemia needing blood transfusion, for whom blood and bone marrow transplant is suitable, but no matching donor is available.
Consequently, Vertex announced a reimbursement agreement with NHS England making the treatment available beginning today. Casgevy will be accessible through the Innovative Medicines Fund for up to 460 eligible patients, as per NICE.
The NICE decision came after an independent committee heard statements on how TDT impacts the lives of patients. TDT is a life-threatening inherited disease in which genetic mutation reduces or prevents production of healthy red blood cells and haemoglobin.
The condition often manifests as intense fatigue; infants may suffer delayed development, misshapen bones, and enlarged organs. Treatment involves lifelong blood transfusions and iron chelation therapy.
Stem cell transplant is a potentially curative option, but is limited by matched donor scarcity. This is especially true for people of South Asian ethnicity who are disproportionally affected and for whom matching donors are particularly rare, according to Dr. Diana Hernandez, a group leader at the medical nonprofit Anthony Nolan.
Hernandez added that Casgevy also has potential against sickle cell disease – “Sickle cell largely affects patients from African and African-Caribbean backgrounds, who are less likely to have a matching stem cell donor, and this community has been waiting years for a new, effective treatment.”
The Medicines and Healthcare products Regulatory Agency (MHRA) granted Casgevy the world-wide first authorisation for a CRISPR-based therapy in November 2023. The approval was for both TDT and sickle cell disease. At a list price of £1.65mn (~$2mn) per course, this reimbursement agreement has significantly increased availability for patients in England.
Casgevy is a one-time therapy in which a patient’s haematopoietic stem cells are removed and edited through clustered regularly interspaced short palindromic repeats (CRISPR) technology that uses the Cas9 enzyme at the BCL11A gene. Modified cells are transfused into the patient, increasing foetal haemoglobin production in red blood cells without the risk of rejection.
Vertex is also working with reimbursement authorities in the EU to make their therapy available to TDT and SCD patients, as has been done in the US, Saudi Arabia, and Bahrain. The company is poised to compete with bluebird Bio’s CRISPR-based SCD therapy Lyfgenia (lovotibeglogene autotemcel).
