Gene Therapy Approved for sickle cell disorder
Casgevy will be available on the NHS for treating sickle cell disorder.
31 January 2025
We are delighted to let you know that NICE has today approved the groundbreaking gene therapy, Exa-cel, or Casgevy, for use through the NHS in people living with sickle cell disorder.
We will update you on the details as soon as we’ve had a chance to fully absorb the documents just released by NICE (National Institute for Health and Care Excellence). For now, we wanted to share the fantastic news.
Our Chief Executive, John James, had this to say:
“We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. The significance of this milestone for the sickle cell community cannot be understated. It was achieved after we staged a long and determined campaign, and today’s result will give hope to many.
We extend our heartfelt thanks to the incredible individuals who shared their personal stories, challenges, and experiences of sickle cell as part of the campaign. Their courage and advocacy played an important role in bringing this new gene therapy treatment to fruition.
While today’s news is incredible, we remain acutely aware that not everyone with sickle cell will be eligible for the potentially life-changing benefits of Casgevy. Furthermore, this new therapy joins a disappointingly limited range of treatments currently available for this complex and challenging condition.
There is still much work ahead to ensure that everyone living with sickle cell has access to the care, treatments, and support they deserve. We remain dedicated to our commitment to advocating for improved treatment options and better outcomes for all those affected by this condition.
For now, we take a moment to celebrate this historic milestone—a turning point in the fight against sickle cell that promises to change lives for the better.”
See the documents on the NICE website : https://www.nice.org.uk/guidance/indevelopment/gid-ta11249