Vertex In CASGEVY Reimbursement Deal With NHS England For Sickle Cell Disease Patients

Vertex Pharmaceuticals

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(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX), a commercial-stage global biotechnology company, announced Friday a reimbursement agreement with NHS England for eligible sickle cell disease or SCD patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY (exagamglogene autotemcel).

According to the firm, the reimbursement agreement comes as the National Institute for Health and Care Excellence or NICE issues positive guidance recommending CASGEVY's use in the NHS.

With this, eligible SCD patients in England now have access to the therapy following the prior agreement for transfusion-dependent beta thalassemia or TDT patients announced last August.

SCD is a debilitating, progressive, life-shortening genetic disease, and SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization.

CASGEVY, a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy, is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world.

The Conditional Marketing Authorization in Great Britain for CASGEVY is for the treatment of patients 12 years of age and older with either TDT or SCD.

Ludovic Fenaux, Senior Vice President, Vertex International, said, "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."

Vertex said it is continuing to engage with experienced hospitals throughout England to establish a network of independently operated authorized treatment centers or ATCs, as the administration of the therapy requires experience in stem cell transplantation and the management of hemoglobinopathies.

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