UNITED KINGDOM – The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted conditional approval for Casgevy, a revolutionary CRISPR-based gene-editing therapy jointly developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

This landmark decision represents the world’s first-ever authorization of a CRISPR-based gene-editing therapy and stands as a watershed moment in the realms of science and medicine, as expressed by Vertex CEO Reshma Kewalramani.

Casgevy, an autologous CD34+ cell-enriched population genetically modified through CRISPR/Cas9, targets sickle cell disease (SCD) and transfusion-dependent beta thalassaemia (TDT).

The conditional approval applies to eligible patients aged 12 and older with SCD experiencing recurrent vaso-occlusive crises (VOCs) or TDT when an HLA-matched related haematopoietic stem cell donor is not available.

The MHRA’s decision rests on compelling evidence from the CLIMB-111 and CLIMB-121 studies, where positive outcomes underscored Casgevy’s efficacy.

Notably, in the SCD trial, 97% of patients experienced a profound reduction in severe pain crises for at least 12 months post-treatment.

Meanwhile, the TDT study demonstrated that 93% of participants did not require a red blood cell transfusion for a similar post-treatment period, with additional patients showing a substantial (>70%) reduction in transfusion needs.

While the therapy awaits evaluation in the European Union, the U.S. Food and Drug Administration (FDA) is expected to announce a decision on SCD approval by December 8.

Julian Beach, the MHRA’s interim executive director of healthcare quality and access, has stressed the ongoing vigilance over Casgevy’s safety and effectiveness.

The agency will meticulously track real-world safety data and post-authorization safety studies conducted by the manufacturer.

Amid the groundbreaking approval, the issue of pricing emerges as a crucial consideration. An FDA advisory panel’s agreement to assess potential safety risks post-approval has been accompanied by Vertex’s proposal of a comprehensive 15-year follow-up plan.

An analysis by the Institute for Clinical and Economic Review (ICER) in the U.S. suggested that a cost of up to US$1.9 million would be deemed cost-effective for Casgevy and Bluebird bio’s gene therapy lovo-cel in the context of SCD.

With an estimated 2,000 eligible patients in the UK, Vertex is actively engaging with national health authorities to ensure expeditious access to Casgevy.

As the UK heralds the green light for this pioneering gene-editing therapy, it not only signifies a historic moment but also exemplifies a significant leap forward as CRISPR-based technologies solidify their presence in the commercial healthcare landscape.

This revolutionary development underscores the transformative potential of gene-editing therapies, offering renewed hope to individuals grappling with challenging genetic conditions. 

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