NICE recommends gene therapy for severe sickle cell disease

The National Institute for Health and Care Excellence (NICE) has issued positive final draft guidance for a one-off gene therapy which provides a potential cure for certain patients with severe sickle cell disease.

This approval of CASGEVY^® (exagamglogene autotemcel) is indicated for individuals 12 years and older who have certain types of severe sickle cell disease.

Vertex Pharmaceuticals’ CRISPR/Cas9 gene-edited therapy will be available under a managed access scheme for this indication, a decision which is a reversal of NICE’s previous draft recommendation in March 2024, Professor Laurence Hurst, Professor of Evolutionary Genetics, The Milner Centre for Evolution, University of Bath explained. The treatment received conditional European approval at the beginning of 2024. 

At present, the only curative treatment for the disease is a donor stem cell transplant. Availability of exa-cel on the NHS means patients have a treatment option when a stem cell transplant is suitable but no matched donor is available.

“Exa-cel is potentially a life-long cure – the patients can make their own non [sickle cell disease] inducing blood, thus immune rejection should not be an issue,” commented Professor Hurst.

This evaluation of CASGEVY for use on the NHS included “more uncertainty in the evidence and a higher cost-effectiveness estimate than NICE normally considers to be value for money for the NHS”. The regulatory body explained this was due to considering potential health inequalities. For instance sickle cell disease is more common in individuals from African, Caribbean, Middle Eastern or South Asian family backgrounds.

Increasing gene therapy access in the UK to treat blood disorders

This decision by NICE follows the prior agreement for transfusion-dependent beta thalassemia announced in August 2024, Vertex confirmed.

“We are pleased to have reached this new agreement that ensures both eligible [sickle cell disease] and [transfusion-dependent beta thalassemia] patients can now be treated with CASGEVY, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system,” commented Ludovic Fenaux, Senior Vice President, Vertex International.

“Excitingly, advances in gene editing are happening very rapidly at the moment and it seems likely that cheaper, safer and more effective forms of gene editing will emerge for sickle cell disease over the coming years, offering the prospect of a curative treatment which is universally applicable, even in low income countries where the majority of patients live,” Professor David Rees, Professor of Paediatric Sickle Cell Disease, King’s College London shared.

NICE stated that additional data will be collated from patients receiving CASGEVY on the NHS, prior to NICE’s evaluating the medicine further. This extra evidence received through the managed access agreement could help mitigate remaining uncertainty of the treatment’s impact.