Insider Insights

World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia


The Medicines and Healthcare products Regulatory Agency (MHRA) granted a conditional marketing authorisation to Casgevy (exagamglogene autotemcel), a CRISPR/Cas9 gene-editing therapy for the treatment of sickle cell disease (SCD) and transfusion-dependant beta thalassaemia (TDT). This marks the world’s first regulatory approval of a medicine using CRISPR gene editing.

Casgevy had been awarded an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) as a result of its addressal of significant unmet medical need. Clinical trials for Casgevy “met their respective primary outcome of becoming free from severe vaso-occlusive crises or transfusion independent for at least 12 consecutive months” as noted by Vertex, a co-developer for Casgevy.

Julian Beach, Interim Executive Director of Healthcare Quality and Access at the MHRA said: “I am pleased to announce that we have authorised an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent β -thalassaemia, relieving the symptoms of disease.”

Casgevy has been estimated to be eligible for 2,000 individuals in the UK aged 12 years or older, who are unable to receive a stem cell transplant from a matched donor. This scientific milestone comes a decade after researchers first described how CRISPR could be utilised to alter DNA in genetic medicine.

Stay in the know, subscribe to our newsletter

Be the first to receive exclusive content on the latest from the pharmaceutical and market access sector.