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Hemgenix (etranacogene dezaparvovec) is now available on the European market for the treatment of severe and moderately severe haemophilia B in adults without factor IX inhibitors (autoantibodies produced by the immune system that make drugs against factor IX less effective).
The recommendation for the granting of marketing authorization in EU countries is from the EMA.
The recommendation for the granting of marketing authorization in EU countries is from the EMA.
Brussels, 16 December 2022
Hemophilia B is an inherited disorder characterized by an increased bleeding tendency due to a partial or complete deficiency of coagulation factor IX, a protein needed to produce blood clots to stop bleeding.
Factor IX deficiency is the result of mutations in the respective coagulation factor gene. Prolonged bleeding episodes in patients with hemophilia B can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain. Hemophilia B is a rare, debilitating disease that affects approximately 1 newborn in 20,000 to 50,000 males alive.
Factor IX deficiency is the result of mutations in the respective coagulation factor gene. Prolonged bleeding episodes in patients with hemophilia B can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain. Hemophilia B is a rare, debilitating disease that affects approximately 1 newborn in 20,000 to 50,000 males alive.
Currently, medicines licensed for the treatment of haemophilia B are aimed at preventing bleeding episodes or treating bleeding episodes that can occur during surgery or in an emergency even when patients are on regular medication. Patients require a routine lifelong treatment regimen of intravenous infusions of factor IX replacement products to maintain sufficient factor IX levels. Therefore, there is an unmet medical need for novel therapeutic approaches that could free patients from the burden of frequent or episodic infusions at the time of a bleeding event.
Hemgenix is the first gene therapy for the treatment of haemophilia B. It is given as a single infusion. Etranacogene dezaparvovec, the active substance in Hemgenix, is based on a virus (adeno-associated virus or AAV) that has been modified not to cause disease in humans. The virus contains copies of the gene responsible for making factor IX. When injected into a patient's vein, the virus is transported to the liver where the gene is taken up by the patient's liver cells and starts producing factor IX, thus limiting bleeding episodes.
Read more....
Hemgenix is the first gene therapy for the treatment of haemophilia B. It is given as a single infusion. Etranacogene dezaparvovec, the active substance in Hemgenix, is based on a virus (adeno-associated virus or AAV) that has been modified not to cause disease in humans. The virus contains copies of the gene responsible for making factor IX. When injected into a patient's vein, the virus is transported to the liver where the gene is taken up by the patient's liver cells and starts producing factor IX, thus limiting bleeding episodes.
Read more....
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Source: EMA - © EMA [1995-2023]
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