Marisa Wexler, MS,  senior science writer—

Most people with hemophilia A on preventive treatment with Hemlibra (emicizumab) will experience spontaneous or traumatic bleeds at some point, according to a study using real-world data from Israel. Results also indicated the risk of spontaneous bleeds while on Hemlibra is higher for older individuals. “Our findings indicate that…

Preventive treatment with Hemlibra (emicizumab) safely and effectively reduced bleeding rates in children with hemophilia A who were treated at a center in Texas, according to a new report. “We demonstrate a successful experience with emicizumab prophylaxis and safe [surgical] approach with a focus on minimizing postoperative bleeding,”…

Two pharmaceutical companies, LeaderMed Health Group and OPKO Health, are teaming up to develop, manufacture, and commercialize two experimental medications in China and other locations in Asia. One of the investigational medicines, Factor VIIa-CTP, is being developed as a potential treatment for hemophilia. “Through this joint venture, we…

The U.S. Food and Drug Administration (FDA) has restored its approval of BeneFIX as a routine prophylactic, or preventive, treatment for bleeds in children and adolescents under 16 with hemophilia B. Marketed by Pfizer, BeneFIX is a lab-made version of factor IX (FIX), the blood clotting protein that…

Preventive treatment with marzeptacog alfa activated (MarzAA) can reduce bleed frequency substantially in men with hemophilia A or B who developed inhibitors to conventional replacement therapies, according to data from a Phase 2 clinical trial. The findings were reported in the study, “Subcutaneous engineered factor…

Treatment with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) can reduce bleeding frequency and the use of replacement therapies, while maintaining or improving the quality of life of people with severe hemophilia A for up to five years, updated Phase 1/2 trial data show.  “These…

Effectiveness in controlling bleeds and the potential for less frequent use of a treatment are two priorities people with hemophilia place on a potential gene therapy, and both ranked slightly higher than safety, a survey study of patients in the U.S. reports. The study, “Patient preferences and…

People with severe hemophilia A who switch from standard to extended half-life replacement therapies experience fewer bleeds and require less frequent infusions, an analysis of patients in Austria indicates. Whether these gains are reflected in lower overall treatment costs, however, is less clear, its researchers added. Data from this…

Spending on treatments for hemophilia by Medicaid, a U.S. government health insurance program for select groups, more than tripled from 2005 to 2019, an analysis reported. Its researchers expect this finding will help state agencies with decisions regarding treatment coverage based on their Medicaid budget, given that the program…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…