Patricia Inácio, PhD,  science writer—

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…

A new analysis of two Phase 3 clinical trials confirms that extending preventive treatment with Alprolix is safe and leads to markedly decreases in bleeding episodes of severe hemophilia B patients. The results were recently presented at the 59th Annual Meeting of the American Society of Hematology (ASH) in…

Injection of Hemlibra (emicizumab-KXWH) every four weeks helps control bleeding in hemophilia A patients, an interim analysis of the Phase 3 HAVEN 4 study shows. The results, announced by Genentech, are in agreement with several previous clinical trials – HAVEN 1 (NCT02622321),…

Swedish Orphan Biovitrum (Sobi) announced the successful enrollment of the first hemophilia A patient in the company’s ReITIrate clinical trial. The Phase 4 study (NCT03103542), sponsored in collaboration with Bioverativ Therapeutics, is actively recruiting participants who have developed inhibitors and who have failed to respond to other therapies. Researchers will…

Pfizer Rare Disease has developed a new video game and wearable wristband to help hemophilia patients learn more about their condition and promote activity tracking in the U.S. The video game, Hemocraft, and the HemMobile Striiv Wearable wristband were launched at the National Hemophilia Foundation’s 69th Annual Meeting in Chicago, Aug. 24-26.

The U.S. Food and Drug Administration (FDA) has granted priority review to the therapy emicizumab for patients with hemophilia A with factor VIII inhibitors, Genetech, the drug’s developer, announced. The FDA also announced the acceptance of Genetech’s Biologics License Application (BLA) for emicizumab prophylaxis (preventive) therapy in adults, adolescents and children…

Information from patient surveys can help doctors do a better job of managing hemophilia and improving patients’ outcomes, an American study reports. The research, which appeared in the journal Patient Preference and Adherence, was titled “Construct validity of patient-reported outcome instruments in US adults with hemophilia: results from the Pain, Functional…