المملكة: The Food and Drug General Authority adopts the first genetic treatment for hemophilia “B” patients in the Kingdom

The Food and Drug Authority has announced the registration of the hemgenix, to be the first approved genetic treatment for medium and very dangerous hemophilia in the Kingdom. Himophilia B is a life -threatening disease, a rare genetic disorder caused by a gene that is responsible for the production of the ninth “IX” clot, a protein that plays a fundamental role in the process of blood clotting and stopping bleeding.
The effectiveness, safety and quality of the product
The authority explained that the preparation was recorded after its evaluation accurately in terms of its effectiveness, safety, quality, and its fulfillment of the required standards, as this product relies on the technique of genetic therapy using the “AAV” viral carrier, which contains copies of the gene responsible for the production of the ninth “IX”.
The method of use
After injecting the drug in the vein, the body begins to produce the ninth clotting factor, which contributes to restoring the blood clotting process and stopping the bleeding naturally. The drug is used only once to stimulate the production of the ninth “IX” clot.
Non -serious side effects
She noted that the clinical studies of the drug showed that there were non -serious side effects, the most common headache, increased liver enzyme levels in the blood, and similar symptoms of influenza. These studies also resulted in the presence of drugs with some drugs that have an effect on liver function.
Quality therapeutic options
It is noteworthy that the registration of this product comes as an extension of the role of the “Authority” in the field of enhancing the availability of qualitative treatment options for patients in the Kingdom of Saudi Arabia, especially those that depend on biotechnology applications, which are witnessing an accelerated scientific development. To stimulate the polarization of these innovative drugs, the authority launched a promising drug recording program that gives them priority in evaluation and registration.