NICE recommends Casgevy for treatment of severe beta-thalassaemia

A new gene therapy, Casgevy, has been recommended for use on the NHS in England for people living with a severe form of beta-thalassaemia.

Beta-thalassaemia is an inherited blood disorder caused by a genetic mutation that affects production of healthy red blood cells and haemoglobin – the protein that carries oxygen around the body.  The CRISPR-based gene therapy has been approved by the National Institute for Health and Care Excellence (NICE), meaning that about 460 patients currently living with transfusion-dependent beta thalassaemia could be eligible for the treatment.

'We are committed to reducing healthcare inequalities by rolling out new and pioneering treatments on the NHS for conditions, such as thalassaemia, which disproportionally affects people from some minority ethnic backgrounds,' said Professor Bola Owolabi, director of the national healthcare inequalities improvement programme at NHS England.

'We hope this ground-breaking treatment enables people with beta thalassaemia to live longer, more independent and higher quality lives – without the need for regular treatments and hospital appointments and without pain, fatigue and other side effects that can come with this severe and life-limiting condition,' she added.

Casgevy works by editing a gene in a patient's bone marrow stem cells so that the body can produce functional haemoglobin. The patients' own blood stem cells are collected and modified outside the body before being transplanted back into the patients. However, patients must firstly undergo chemotherapy and radiotherapy to wipe out their immune system before it is rebuilt in a single-dose infusion of their own modified stem cells.

The drug carries a list price of US$2 million per patient, but the NHS negotiated a confidential discount.

'We're delighted with this historic decision to approve the UK's first ever CRISPR-based therapy. Casgevy offers an effective cure for transfusion-dependent beta thalassaemia – a debilitating condition that was previously incurable in patients who don't have a stem cell donor,' said Yasmin Sheikh, head of policy and public affairs at stem cell charity Anthony Nolan.

The UK Medicines and Healthcare products Regulatory Agency approved Casgevy for use in beta-thalassaemia and sickle cell disease in the UK in November 2023 (see BioNews 1216). However, NICE are still negotiating the use of Casgevy for sickle cell disease, another inherited blood disorder.