Pfizer said it will stop development and commercialization of hemophilia B treatment Beqvez (fidanacogene elaparvovec-dzkt). “Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies…
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Total hip replacement surgery for hemophilia patients is usually successful in creating a more functional joint, according to a systematic review study. However, clinicians and patients need to be aware of potential risks, such as infection and bleeding. “This paper confirms the findings of previous studies that [[total hip replacement…
Weekly and monthly treatment with Mim8, an investigational antibody therapy, was seen to be better than on-demand or standard preventive treatments at reducing bleeding episodes in patients with hemophilia A, regardless of inhibitor status, who took part in a clinical trial. Top-line results from the global Phase 3…
Altuviiio (efanesoctocog alfa) reduced bleeding and was safe for boys and young men with severe hemophilia A, according to a recent study. Researchers also reported an increase in participants’ confidence in everyday activities and no return of neutralizing antibodies that may inhibit treatment, even in patients who are usually…
Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…
A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…
Patients with moderate to severe hemophilia A who switched to Kovaltry (octocog alfa) showed no worsening of their annual bleeding rate, according to real-world data from a Phase 4 study. Adhering to the therapy remained high and patients reported high treatment satisfaction. Overall, the study “confirms and extends clinical trial…
In the U.S., hemophilia A is estimated to affect one in every 5,000 male births, hemophilia B one in 25,000 male births, and hemophilia C one in every 100,000 people (males and females). Hemophilia A is thus four times more common than hemophilia B, and about 10 times more common than…
AMT-060 was a first-generation gene therapy for hemophilia B that was tested in clinical trials as a potential treatment for reducing the risk of bleeding episodes in patients.
The hemophilia B treatment Rebinyn is available in the United States, its maker Novo Nordisk reports. Rebinyn, which is administered intravenously, replaces the blood clotting factor IX that is missing in the disease. The U.S. Food and Drug Administration approved it in May 2017 to control bleeding episodes and manage bleeding…