ACCESS TO INNOVATION | UK Greenlights Gene-Editing Therapy Casgevy for β-Thalassaemia Treatment
As of today, August 8, the ground-breaking gene-editing therapy Casgevy has been authorized by the National Institute for Health and Care Excellence (NICE) for use on the NHS England, specifically targeting older children and adults suffering from severe thalassaemia.
This development opens new treatment avenues for an estimated 460 patients aged 12 and above in England who rely on regular transfusions due to β-thalassaemia, leveraging innovative CRISPR gene-editing technology.
The NHS has struck a pivotal agreement with Vertex Pharmaceuticals, the manufacturer of Casgevy, ensuring the therapy’s inclusion in the Innovative Medicines Fund (IMF). This arrangement guarantees expedited access for NHS patients while compiling comprehensive data on its long-term benefits over the next five years. Although the therapy’s official price tag stands at £1,651,000 ($2 million), it will be available at a confidentially discounted rate.
NICE’s committee determined that Casgevy “could represent a potential cure for some people with transfusion-dependent β-thalassaemia, freeing them from the burden and risks associated with regular blood transfusions,” as stated Helen Knight, NICE’s Director of Medicines Evaluation.
In the US, Casgevy was approved by the FDA in December 2023 for the treatment of sickle cell disease and in January 2024 for the treatment of transfusion-dependent β-thalassaemia following an October FDA advisory committee vote. Since then, approximately 20 patients have embarked on their treatment journeys beginning with cell collection, with Vertex establishing over 35 active treatment centers worldwide. Already, around 35,000 patients across the US and Europe have been identified as potential candidates for this revolutionary therapy.
One early beneficiary of this pioneering treatment is Abdul-Qadeer Akhtar from Hemel Hempstead, UK, who participated in a clinical trial in 2020. Reflecting on his experience, he shared: “My overall health and quality of life have significantly improved. I am gaining more from my workouts and have even taken up boxing. I can travel more freely now, which is fantastic – I am eager to embrace life to the fullest.”
Dr. Androulla Eleftheriou, Executive Director of the Thalassaemia International Federation (TIF), commented, “This marks a significant leap forward in thalassaemia treatment, with the potential to dramatically improve the lives of eligible patients. Casgevy’s approval highlights the transformative power of medical innovation, setting the stage for even more revolutionary curative solutions in the years ahead.”
Source: NHS England
