BRIEF—Vertex’ Cas9 gene-edited therapy Casgevy approved in Saudi Arabia

US drugmaker Vertex Pharmaceuticals has received marketing approval from the Saudi Arabia’s Food and Drug Authority (SFDA) for its Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Casgevy is approved for the treatment of people 12 years of age and older with SCD or TDT.

The Kingdom of Saudi Arabia has among the highest prevalence rates of SCD and TDT in the world, with thousands of patients living with these genetic blood disorders, according to Vertex’ statement.

Last November, Vertex made history by gaining approval for the first CRISPR-based drug, Casgevy from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) followed in December by US Food and Drug Administration (FDA) authorization,

Also in December, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the conditional approval of Casgevy.