Patricia Inácio, PhD,  science writer—

Data from ongoing Phase 3 clinical trials show that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis — or preventive treatment — greatly outperforms current standard-of-care therapies, leading to significant reductions in the number of bleeds in hemophilia A patients regardless of inhibitor status. Promising results from these pivotal studies may lead…

A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies has dosed its first participant. “Administration of valoctocogene roxaparvovec to this first patient seropositive for the AAV5 capsid is an important next step in our plan to…

Teaching hemophilia patients who have undergone joint replacement surgery how to administer their intravenous (IV) self-infusions in sterility, can decrease the rate of infections post-surgery significantly, a six-year education program shows. The program outcomes will be shared at the upcoming World Federation of Hemophilia 2018 World Congress May 20-24…

Hemlibra (emicizumab) is an effective and less costly therapy for hemophilia A patients who have substances in their blood that prevent clotting factor therapies from working as well as they should, according to a therapy rating organization. The Institute for Clinical and Economic Review (ICER) updated an initial report on Hemlibra…

Eloctate use in immune tolerance induction (ITI) therapy shows promise in high-risk patients with severe hemophilia A and inhibitors treated for the first time, Bioverativ announced. The results support a potential benefit for some patients who tried and failed ITI with other factors. The study, “Recombinant factor VIII…

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…

Gene therapy company uniQure released new data showing that its lead therapy AMT-060 for severe and moderately severe hemophilia B is safe and effective for up to two years, with adults requiring fewer doses of replacement therapy and showing a marked decrease in the spontaneous bleeding rate. The results support uniQure’s clinical…

Among its corporate goals for 2018 announced recently, Alnylam Pharmaceuticals said it will continue to clinically develop the investigational RNA therapy fitusiran to treat hemophilia. Fitusiran, also known as ALN-AT3SC, is an RNAi therapeutic agent that targets a protein called antithrombin — which inhibits blood clotting — while increasing production of the…

Catalyst Biosciences has amended the protocol of its ongoing Phase 1/2 clinical trial investigating the highly potent recombinant human factor IX variant CB 2679d in previously treated hemophilia B patients. The amendment has shortened the study’s duration, which is a dose-escalation study to investigate the safety and drug and organism responses (pharmacokinetics and…

The U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, an investigational therapy for hemophilia, Alnylam Pharmaceuticals announced. “We are pleased with the FDA’s decision to lift the clinical hold, as fitusiran holds the potential to help improve the lives of people…