SAUDI ARABIA – Vertex Pharmaceuticals Incorporated has earned approval from the Saudi Food and Drug Authority (SFDA) for its groundbreaking CRISPR/Cas9 gene-edited therapy, Casgevy.

In a significant milestone in the medical world, the Saudi Food and Drug Authority has approved Casgevy (exagamglogene autotemcel) as a treatment for two genetic blood disorders.

The authorization represents a major advancement in the treatment of two genetic blood disorders, namely sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

In a publication of BNN Breaking, an independent news network, Vertex Pharmaceuticals said: “The company is striving to expedite patient access to the therapy by ensuring Casgevy is incorporated into hospital formularies, facilitating reimbursement and availability.”

The company highlighted that Casgevy utilizes CRISPR/Cas9 technology to modify patients’ hematopoietic stem cells, allowing for precision editing of DNA to rectify genetic mutations. 

The authorized therapy works by editing the genetic mutation in the affected gene, enabling the body to produce hemoglobin appropriately,” stated Vertex Pharmaceuticals.

Vertex Pharmaceuticals reaffirmed patients that Casgevy will be incorporated into hospital formularies in a move to help streamline the process for patients, making the groundbreaking therapy more accessible to those in need.

To tackle the high prevalence of SCD and TDT in Saudi Arabia, the Ministry of National Guard Health Affairs (MNGHA) has been designated as the initial Authorized Treatment Center (ATC) for administering Casgevy.

Vertex Pharmaceuticals is actively working to expand the network of ATCs to include other hospitals, such as the King Faisal Specialist Hospital (KFSH).

On the landmark SFDA approval, Vertex Pharmaceuticals confirmed: “This innovative approach to treatment marks a significant shift in the field of gene therapy, particularly for these hereditary blood disorders.”

It is worth highlighting that the SFDA evaluated the effectiveness, safety and quality of Vertex Pharmaceuticals’ gene-edited therapy, Casgevy, 

The SFDA said that the effectiveness, safety and quality of the treatment was evaluated, after meeting the necessary standards, as the treatment works by genetic editing of the genetic mutation in the affected gene, so that the body can produce hemoglobin properly. 

This is through extracting stem cells from the patient’s bone marrow and genetically editing them in the laboratory, and then it is re-implanted into the patient’s body to give a long-term effect.

Moreover, Vertex Pharmaceuticals’ authorized therapy is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. 

Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology. Patients’ hematopoietic stem cells are modified by genome editing using CRISPR/Cas9 technology.

Taking this into account, the SFDA approval attests to Vertex Pharmaceuticals’ capabilities in bringing complex generics and difficult-to-manufacture products to the Saudi market.

The SFDA conducts regular inspections of facilities under its supervision, in collaboration with relevant authorities, to ensure compliance with technical regulations and food specifications. Moreover, legal action is taken against violators to ensure consumer safety and health.

Following the SFDA approval, Vertex Pharmaceuticals is looking to expand access to the gene-edited therapy, Casgevy, to the Kingdom of Saudi Arabia.

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