The Saudi Food and Drug Authority (SFDA) has approved the first gene therapy for Hemophilia (B) in the Kingdom of Saudi Arabia. It has announced the registration of Hemgenix (etranacogene dezaparvovec) for use in patients with moderate to severe Hemophilia (B), a life-threatening genetic disorder. This condition is caused by a defect in the gene responsible for producing clotting factor IX, a protein essential for effective blood clotting and stopping bleeding.

Efficacy, Safety, and Quality of Hemgenix

The SFDA confirmed that Hemgenix was approved after a rigorous evaluation of its efficacy, safety, quality, and compliance with required standards. The gene therapy utilizes an adeno-associated virus (AAV) viral vector to deliver copies of the gene responsible for producing clotting factor IX.

Method of Administration

Hemgenix is administered as a single intravenous infusion, delivering a functional factor IX gene to prompts the body to produce clotting factor IX. This restored production helps normalize blood clotting and control bleeding.

Non-Serious Side Effects

Clinical studies of Hemgenix revealed some non-serious side effects. The most commonly reported adverse events include headache, increased liver enzyme levels, and flu-like symptoms. Patients should inform their physician of all medications they are taking, as Hemgenix may interact with drugs that affect liver function.

Novel Treatment Option

The registration of Hemgenix reflects the SFDA's ongoing efforts to enhance the availability of advanced treatment options for patients in the Kingdom, particularly in the field of biotechnology. The SFDA recognizes the rapid scientific advancements in this area and is committed to making these innovative medicines accessible. To encourage the development of these therapies, the SFDA launched the Breakthrough Medicine Program, which facilitates the expedited review and approval of such treatments.