News

UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B. In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The…

Two gene therapies from Sangamo Therapeutics for hemophilia A and B have been granted special regulatory designations by the U.S. Food and Drug Administration (FDA). Sangamo said in a press release that the FDA has granted orphan drug status to SB-525  gene therapy for hemophilia A, providing the company…

In recognition of the World Hemophilia Day, BDI Pharma employees company-wide united to raise awareness and money to support those affected by bleeding disorders. Events were held April 17 at the company’s South Carolina, Texas and Kansas sites, with employees joining remotely to raise money to support the WFH (World Federation of Hemophilia) by wearing…

Novo Nordisk expects a decision this month by the U.S. Food and Drug Administration (FDA) on its product N9-GP  (nonacog beta pegol) as a treatment for hemophilia B. Approval of the drug may depend on the conclusions the FDA makes regarding safety concerns raised by its blood products advisory committee,…

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…

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…

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…

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…