England Secures Access to CASGEVY for Sickle Cell Disease Patients Under NHS Agreement

03 February 2025 | Monday | News


Following NICE’s positive guidance, Vertex Pharmaceuticals’ CRISPR/Cas9 gene-edited therapy will now be available to eligible SCD patients, expanding on the prior agreement for beta thalassemia treatment.
Image Source : Public Domain

Image Source : Public Domain

 Agreement means eligible sickle cell disease (SCD) patients in England now have access to CASGEVY -

- Agreement for CASGEVY in transfusion-dependent beta thalassemia (TDT) was previously reached in August 2024 -

Vertex Pharmaceuticals announced  a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel).

The reimbursement agreement comes as the National Institute for Health and Care Excellence (NICE) issues positive guidance recommending CASGEVY’s use in the NHS. It means that eligible SCD patients in England now have access to the therapy following the prior agreement for transfusion-dependent beta thalassemia (TDT) patients announced last August.

“Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease,” said Ludovic Fenaux, Senior Vice President, Vertex International. “We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with CASGEVY, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system.”

The administration of the therapy requires experience in stem cell transplantation and the management of hemoglobinopathies; therefore, Vertex is continuing to engage with experienced hospitals throughout England to establish a network of independently operated authorized treatment centers (ATCs).

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