News

UniQure‘s investigational gene therapy AMT-060 for severe hemophilia B has received PRIME designation by the European Medicines Agency (EMA). The EMA designation was based on the results of a Phase 1/2 open-label, dose-escalating study of AMT-060 (NCT02396342). The study included 10 patients, each receiving…

More than 12,300 hemophilia cases in developing countries were treated with therapy donations from Bioverativ and its partner Sobi in 2016, Bioverativ said in a press release about its participation in World Hemophilia Day. The company, which is promoting genetic testing in women and girls who could be susceptible to bleeding episodes, worked with other…

Interim results of Phase 3 clinical studies investigating two major challenges in hemophilia — the development of inhibitors (antibodies to the drug used to treat bleeding episodes) and the need for repeated venous injection of blood clotting protein Factor VIII (FVIII) — were recently released. The results were presented by…

First results from the Phase 3 HAVEN 2 study evaluating the effectiveness and safety of emicizumab in children younger than 12 with hemophilia A look promising. Roche announced that preventive treatment with emicizumab caused a reduction of the number of bleeds over time, with no major adverse events reported so…

Novo Nordisk A/S has launched HemaGo XChange, a new web-based service portal for people with bleeding disorders. This platform will address the overwhelming demands that patients with hemophilia might face in the monitoring and sharing of information about their disease. HemaGo XChange allows patients to monitor and share treatment…

Ixinity (recombinant factor IX product, IB1001) was found to be safe and effective in previously treated children with hemophilia B younger than 12, according to data from Aptevo Therapeutics‘ ongoing Phase 3 clinical trial. The company presented data from the analysis of two studies at the recent Hemostasis and Thrombosis…

San Francisco-based Catalyst Biosciences has completed toxicology studies of its new drug CB 2679d/ISU304 — a highly potent next-generation coagulation factor to treat hemophilia B. This  follows the therapy’s recent Investigational New Drug (IND) approval, a necessary step before starting clinical studies, by the Korean Ministry of Food and Drug Safety. Patients…

The lives of four inspiring patients with bleeding disorders were introduced at the “Hope Ignites: Stories to Inspire” patient program at the Hemophilia Federation of America (HFA) annual symposium recently in Providence, Rhode Island. Sponsored by Octapharma USA, the “Hope Ignites” symposium was for patients with hemophilia to offer hope…

All of the hemophilia B patients in an ongoing Phase 1/2 clinical trial of SPK-9001 experienced clotting-factor increases and reductions in bleeding episodes, according to the therapy’s maker, Spark Therapeutics. University of Pennsylvania medical researcher Adam Cuker will present the updated preliminary results today at the Hemostasis and Thrombosis Research Society (HTRS) 2017…

Dimension Therapeutics’ gene therapy candidate drug DTX101 shows promising results by improving the levels of blood clotting factor IX (FIX) in adult patients with moderate/severe to severe hemophilia B, the company said. Hemophilia B is caused by FIX deficiency, due to mutations on the gene that encodes this protein. Treating…