A new, modified version of recombinant factor VIII (rFVIII), called rVIII-SingleChain, showed great efficacy and safety in treating bleeding events in children with severe hemophilia A. The study, “Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial,” was published in…
Search results for:
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for Afstyla (lonoctocog alfa) for the treatment and prevention of bleeding in adults and children with hemophilia A. Developed by CSL Behring, Afstyla is a single-chain recombinant human factor VIII…
Approximately 30,000 males have hemophilia in the U.S., according to estimates from a recent study, which also found that patients with hemophilia are generally young and that the disease is more common in Northeastern and Midwestern states. The study, “Occurrence rates of haemophilia among males in…
Voncento — a highly concentrated mixture of Von Willebrand factor (VWF) and clotting factor VIII (FVIII) — was seen to safely and effectively treat or prevent bleeds in children, age 12 and younger, with severe hemophilia A, according to data from a Phase 3 trial. Findings were reported in the…
Catalyst Biosciences recently announced updates on two of its lead compounds — Factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) and Factor IX (FIX) dalcinonacog alfa (DalcA) — for treatment of hemophilia A or B with inhibitors, and for hemophilia B. DalcA is a potent recombinant (artificial) Factor IX prophylactic therapy designed to prevent acute bleeding…
In boys and men with severe hemophilia A, starting prophylaxis — preventive treatment given regularly to reduce bleeding — early and maintaining regular physical activity were each linked to a lower risk of hemophilic arthropathy, a progressive form of joint damage caused by repeated bleeding, according to a real-world…
Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A. They also discovered that hemophilia A…
A single dose of BIVV001 is safe and effectively increases factor VIII (FVIII) activity over one month in men with severe hemophilia A, lasting longer in the body than the replacement therapy Advate, results from the EXTEN-A clinical trial suggest. Gains in half-life seen with BIVV001 “could signal…
The hemophilia A treatment Nuwiq (simoctocog alfa) is safe and effective for patients with severe disease, both when used on demand or as a preventative treatment, researchers determined in a recent report. The researchers, who analyzed available data on Nuwuq, detailed their finding in the article, “Efficacy and…
Two new mutations affecting the production of functional clotting factor VIII were identified in a group of Vietnamese patients with hemophilia A, researchers report. Their findings were described in a letter to the editor, “Mutation characteristic of 103 haemophilia A patients in Vietnam: Identification of novel mutations,”…