Exa-Cel (Casgevy) Gene Therapy Update

Exa-Cel (Casgevy) Gene Therapy Update

Today the National Institute for Health and Care Excellence (NICE) announced their draft decision to reject the CRISPR gene therapy, Exa-cel, as a treatment for sickle cell disorder.

Exa-cel, also known as brand name Casgevy, involves modifying a patient’s own stem cells to treat sickle cell. The treatment was approved by the medical regulator MHRA in November for use in the UK as a treatment for sickle cell.

We have joined forces with the Anthony Nolan charity, and together we are urging NICE and pharmaceutical company Vertex to find a solution so this provisional decision can be reversed without delay.

There is now a period of consultation before a second NICE committee meeting to consider the evidence and comments from stakeholders. We are calling on people with lived experience, and healthcare professionals, to contribute to the consultation and inform NICE’s final recommendation. The deadline to respond is 11 April 2024.

This Exa-cel Factsheet   gives further details, and links to where you can send your comments to contribute to the consultation. We aim to make sure the sickle cell community voice is heard, and that NICE is left in no doubt as to the huge impact that sickle cell has on people’s lives.