NICE Endorses Casgevy for NHS Thalassemia Treatment

Vertex Pharmaceuticals and CRISPR Therapeutics have achieved a significant milestone with their CRISPR/Cas9-based gene-editing therapy, Casgevy (exagamglogene autotemcel). The UK's National Institute for Health and Care Excellence (NICE) has recommended Casgevy as a treatment option for patients aged 12 and above with transfusion-dependent beta thalassemia (TDT). This positive recommendation marks a turnaround from the agency's previous decision in March, where the therapy was not endorsed due to concerns over its effectiveness in sickle cell disease (SCD) and its high cost.

Ludovic Fenaux, Senior Vice President of Vertex International, expressed his enthusiasm, describing the approval as a historic moment for individuals living with TDT, who have long had limited treatment options. The therapy had already been approved by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for both SCD and TDT the previous year. Following this favorable decision from NICE, Vertex has secured a reimbursement agreement with NHS England. This agreement will enable up to 460 eligible patients to access Casgevy through England’s Innovative Medicines Fund (IMF).

NICE has set the list price for Casgevy at £1,651,000 (approximately $2,098,138) per course of treatment. While the earlier draft guidance from NICE raised concerns about the long-term effectiveness and cost-effectiveness of Casgevy, the committee has updated its stance. Helen Knight, the director of medicines evaluation at NICE, acknowledged the uncertainties in the evidence regarding long-term benefits. However, she emphasized that the committee believes Casgevy could potentially offer a cure for some individuals with transfusion-dependent beta-thalassemia.

To ensure the successful administration of this complex gene therapy, Vertex plans to collaborate with experienced hospitals across England. This partnership aims to establish a network of independently operated authorized treatment centers to deliver the therapy effectively.

The trajectory of Casgevy's approval process mirrors that of Pfizer’s SCD gene therapy, Oxbryta (voxelotor). Oxbryta also faced an initial refusal from NICE last year but later secured approval in May this year after a price reduction.

Overall, the recommendation of Casgevy by NICE represents a significant advance in the treatment landscape for patients with transfusion-dependent beta thalassemia. This gene-editing therapy provides a new ray of hope for those who have had limited treatment options, potentially transforming their quality of life.