CASGEVY treatment involves editing a patient’s own hematopoietic stem and progenitor cells at the erythroid-specific enhancer region of the BCL11A gene. Credit: CDC on Unsplash.

Vertex Pharmaceuticals has announced the approval of CASGEVY (exagamglogene autotemcel [exa-cel]), the first CRISPR/Cas9 gene-edited therapy for treating sickle cell disease (SCD) and transfusion-dependent beta-thalassaemia (TDT) in patients aged 12 and above.

The company received approval from the Saudi Food and Drug Authority (SFDA), making CASGEVY the first medicine evaluated through the SFDA’s breakthrough medicines programme.

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CASGEVY treatment involves editing a patient’s own haematopoietic stem and progenitor cells in the erythroid-specific enhancer region of the BCL11A gene through a precise double-strand break.

This will result in the production of high levels of foetal haemoglobin in red blood cells.

The therapy has demonstrated the potential to reduce or eliminate vaso-occlusive crises in SCD patients and lessen transfusion requirements in TDT patients.

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Vertex president and CEO Reshma Kewalramani stated: “This approval adds to the list of firsts for CASGEVY. It represents the first medicine ever to receive SFDA breakthrough designation and be approved through this pathway, and Vertex’s first-ever regulatory approval in the Kingdom of Saudi Arabia.

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“Most importantly, with this approval, people living with sickle cell disease or transfusion-dependent beta-thalassaemia have the possibility of a one-time transformative therapy for their disease.”

The Ministry of National Guard Health Affairs is the first authorised treatment centre (ATC) in Saudi Arabia. Vertex is working to qualify more hospitals as ATCs, including the King Faisal Specialist Hospital, to make CASGEVY accessible to patients.

SCD is a genetic condition that causes severe pain and organ damage due to abnormal red blood cells while TDT is a life-threatening disease that requires lifelong blood transfusions and iron chelation therapy.

Saudi Arabia has among the highest global prevalence rates of both SCD and TDT.

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https://news.vrtx.com/news-releases/news-release-details/vertex-announces-approval-first-crisprcas9-gene-edited-therapy

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