Casgevy gene-editing therapy now approved in Saudi Arabia for SCD

Approval for patients 12 and older comes 1 month after similar decision in US

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Saudi Arabia’s regulatory authorities have approved Casgevy (exagamglogene autotemcel), a one-time gene-editing therapy, to treat sickle cell disease (SCD) in adults and adolescents, ages 12 and older.

The approval by the Saudi Food and Drug Authority (SFDA), the country’s equivalent to the U.S. Food and Drug Administration, also covers transfusion-dependent beta thalassemia, a related blood disease.

Developed jointly by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is approved in the U.S. to treat people with SCD who experience recurrent vaso-occlusive crises, known as VOCs. In the U.K., Casgevy is approved under a conditional marketing authorization.

“This approval adds to the list of firsts for Casgevy,” Reshma Kewalramani, MD, Vertex’s CEO and president, said in a press release.

“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,” Kewalramani said.

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Casgevy approval granted via country’s Breakthrough Medicine Program

SFDA’s Breakthrough Medicine Program was launched in 2023 to facilitate and speed the development and review of therapies that address unmet medical needs in people in Saudi Arabia with serious or life-threatening diseases, such as SCD.

The first center certified to provide Casgevy in the country is the Ministry of National Guard Health Affairs, in Riyadh. Vertex is working to certify more hospitals as authorized treatment centers, and to get Casgevy listed on hospital formularies for easier reimbursement.

In SCD, an altered form of hemoglobin — the protein that carries oxygen in red blood cells — is produced, leading to the formation of rigid, sickle-shaped red blood cells that can cause blockages in blood vessels. Beta thalassemia, another blood disorder, results from insufficient production of a specific component of hemoglobin.

In both conditions, the defective hemoglobin leads to low or inadequate levels of functional hemoglobin, causing anemia, or a shortage of healthy red blood cells.

Casgevy uses a gene-editing tool called CRISPR/Cas9 to boost the production of fetal hemoglobin. This is done by modifying the gene that encodes BCL11A, a protein that puts a stop to the production of the fetal form of hemoglobin during normal development, reducing its activity. This enables more fetal hemoglobin to be produced and prevents red blood cell sickling.

The treatment involves collecting a patient’s hematopoietic stem cells — the stem cell precursors that give rise to all mature blood cell types — and editing them in a lab. The cells are then returned to the patient via a stem cell transplant.

According to Vertex, Saudi Arabia has among the highest prevalence rates of SCD and transfusion-dependent beta thalassemia in the world. The approval will provide patients with this conditions “the possibility of a one-time transformative therapy for their disease,” Kewalramani said.