US: HEMGENIX, biologically known as estranacogene dezaparvovec, has been authorized by Heath Canada, according to the Global Biotechnology leader CSL (ASX: CSL). It is to be noted that the medication is the first and only gene therapy for the treatment of Hemophilia B.
According to health experts, estranacogene dezaparvovec is a suitable medication to treat adults with hemophilia B who have to undergo routine prophylaxis. The patients prefer the process of prophylaxis to reduce or prevent the bleeding episodes frequency. It is to be highlighted that no specific experience of HEMGENIX is required for the patients having hemophilia B, ranging from mild to moderate.
The permission is based on a study called HOPE-B, involving 54 participants, and it’s the biggest trial ever done for hemophilia B gene therapy. The important trial results reveal that people with hemophilia B who received one HEMGENIX infusion had their FIX activity levels go up by a lot – first by 36.9% at 18 months and then staying at 36.7% at 24 months after the treatment, compared to the six months before. Between seven and 18 months after the infusion, the average number of bleeds (ABR) went down by 58% compared to the six months before (from 4.13 to 1.73).
The research outlined that around 96 percent of patients, i.e., 52 out of 24, stopped using prophylaxis and discontinued their therapies. According to the reports, in the phase 2b and phase 3 trials, the most common side effects reported were higher ALT and AST levels in the blood, along with symptoms like headaches and flu-like illness.
The Head of Research and Development for CSL – Bill Mezzanott, was quoted saying, “This approval continues to demonstrate CSL’s promise to pursue, develop and deliver new innovative treatment options that meet the needs of the rare disease community.”
He continued, “We believe HEMGENIX has the potential to change the treatment paradigm for people living with hemophilia B as well as the healthcare professionals who treat them, as it addresses the cause of the condition – faulty factor IX gene expression.”
Hemophilia B is a rare and lifelong bleeding disorder caused by a gene defect. This defect leads to a lack of factor IX, which is a protein that helps blood clot properly and is mainly made by the liver.
People with hemophilia B usually receive treatment involving regular infusions of factor IX replacement therapy. This therapy is necessary to temporarily supplement the low levels of blood-clotting factor in their bodies. While these treatments are effective, individuals with hemophilia B must stick to a strict schedule of lifelong infusions.
Despite treatment, people with hemophilia B can still have unexpected bleeding episodes. They may also experience problems like limited mobility, joint damage, or severe pain due to the disease. HEMGENIX offers a way for individuals with hemophilia B to produce their own factor IX, which can reduce the risk of bleeding.
The General Manager Canada, CSL Behring – Philippe Hebert, outlined, “The approval of HEMGENIX in Canada marks an important milestone and we look forward to collaborating with the hemophilia B community to provide access to this innovative treatment option.”
“We are proud to add this treatment to our portfolio of coagulation therapies and look forward to patients benefiting from this therapy,” Hebert outlined.
HEMGENIX got the green light from the US Food and Drug Administration. It also got permission from the European Commission for use in the European Union, the European Economic Area, and the United Kingdom’s Medicines and Healthcare Products Regulatory Agency. The development of HEMGENIX took several years, with uniQure initially leading the clinical trials. Later on, CSL took over the rights to sell this treatment worldwide.