Author Archives: Patricia Inacio PhD

China OKs Hemophilia B Trial Testing BBM-H901 Gene Therapy

Belief Biomed is launching a Phase 1/2 clinical trial in China to test BBM-H901, its investigational one-time gene therapy for hemophilia B. The announcement comes after China’s National Medical Products Administration (NMPA) approved the company’s investigational new drug application, which had been submitted in April, Belief Biomed said in…

EMA OKs Biomarin’s New Application to Approve Roctavian

The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical’s new application seeking approval of its investigational one-time gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. The application was resubmitted to EMA following a request for longer follow-up data from the Phase 3 GENEr8-1 clinical trial (NCT03370913),…

Poorer Joint Health, Greater Pain Evident After COVID Lockdown

Joint pain significantly rose and overall joint health — particularly range of motion — diminished with limitations on physical activities during the COVID-19 lockdown for people with hemophilia and joint disease, a small study from Spain reported. The study “Musculoskeletal Changes in Hemophilia Patients Subsequent to COVID−19…

Study Examines Inhibitor Risk in Non-severe Hemophilia A

Intensive treatment with factor VIII, the clotting protein missing or defective in patients with hemophilia A, is associated with a greater risk of inhibitor development among patients with non-severe disease, a study has found. The findings highlight the importance of closely monitoring these patients in order to prevent or…

Hemophilia B Patients in US Face Great Financial, Personal Burden

Severe hemophilia B patients receiving prophylactic, or preventive treatment with factor IX face a significant financial and personal burden, according to data from two U.S. population-based medical databases. These findings were reported in a study, “Clinical, humanistic, and economic burden of severe hemophilia B in the United States:…