Marisa Wexler, MS, senior science writer —

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…

Refixia or Rebinyn (nonacog beta pegol, or N9-GP), an approved replacement therapy for hemophilia B, is effective and has a good safety profile in previously untreated boys younger than 6 with severe to moderately severe disease, an early analysis of a Phase 3 trial indicates.  In…

CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, can increase factor IX (FIX) levels and significantly reduce bleeding, early studies show. The data, “Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B…

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…

Three hemophilia B patients said they were able to lead a relatively normal life, including being more active and participating in sports, about three years after receiving the investigational gene therapy AMT-060 in a clinical trial. Their experiences were reported in the study, “The Patient…

Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from this open-label study in six patients are expected by June. “We are pleased to have successfully completed the…

The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for Valrox (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s investigational gene therapy for hemophilia A. This is the first time the FDA has accepted an application for a gene…

When administered at a high dose, a gene therapy for hemophilia B known as FLT180a seems able to restore the activity of clotting factor IX (FIX) within a normal range in men with moderate-to-severe disease, early data from Phase 1/2 trial show. The findings were announced in an oral presentation,…

Esperoct (turoctocog alfa pegol) is now available in the U.S. to treat and control bleeding in adults and children with hemophilia A, the therapy’s manufacturer, Novo Nordisk, announced. The treatment’s use was approved by the U.S. Food and Drug Administration (FDA) in February 2019, but its availability…

A modified version of the coagulation protein factor X was able to significantly reduce bleeding in a mouse model of hemophilia A, and may offer a way of treating people with this disease due to factor VIII deficiency, a study suggests. Lab work also showed a potential for treating people…