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.