Once-monthly injection of the investigational RNA therapy Fitusiran reduced the levels of the protein antithrombin, a natural inhibitor of several components of the clotting system, and restored the balance of clotting elements production in adult patients with hemophilia A or B. These findings were featured in the article “Targeting…
Shire has acquired worldwide rights to the antibody-based therapy that Novimmune developed as a treatment for hemophilia A. Geneva-based Novimmune creates therapies for inflammatory diseases, autoimmune diseases and cancer. It uses antibodies that target tumor-associated proteins and the immune checkpoint protein CD47. Shire, which is based in Dublin, is a global…
Hemophilia A patients treated with CSL Behring’s Afstyla (recombinant factor 8 single-chain) require less factor to control bleeding compared to those taking octocog alfa, the company recently announced. The lower consumption holds both for patients using clotting factors on-demand and those who require preventive, or prophylactic treatment. CSL Behring…
The latest results of CSL Behring’s recombinant coagulation factor IX (FIX) Idelvion, also known by its generic name CSL654, demonstrated long-lasting therapeutic potential for both pediatric and adult patients with hemophilia B. The findings were detailed in poster presentations at the 26th Biennial…
Bayer has awarded a combined $2 million in hemophilia research and patient-care grants to 16 scientists and doctors from eight countries. Four of the winners are from the United States. Three represent the Children’s Hospital of Philadelphia and one the Uniformed Services University of the Health Sciences in…
Treatment with Eloctate and Alprolix improves joint health, bleeding episodes and quality of life of patients with hemophilia, Bioverativ and Sweden’s Sobi recently announced. The data is being presented at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress,  through July 13 in Berlin, Germany. Hemophilia is a…
Shire has asked the U.S. Food and Drug Administration to grant Investigational New Drug status to its hemophilia A treatment SHP654. The gene therapy helps generate a clotting protein known as factor VIII that is missing or defective in hemophilia A. Shire said an FDA approval of its request will…
Organizations championing patients with hemophilia and other bleeding disorders have formed a united front to oppose the Better Care Reconciliation Act, the Obamacare replacement legislation that the groups contend will damage the hemophilia community. The National Hemophilia Foundation, Hemophilia Federation of America, Coalition for Hemophilia B and…
European regulators have granted orphan drug status to CB 2679d, a hemophilia B therapy that Catalyst Biosciences developed to prevent bleeding episodes. The therapy, known as ISU304 in South Korea, prevents bleeding by increasing hemophilia B patients’ level of a blood protein called Factor IX, which is deficient in the disease.
Results from two Phase 3 trials support the use of emicizumab for the preventive treatment of hemophilia A in children, adolescents, and adults, Genentech announced. These results will be presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting July 8-13 in Berlin, Germany. Emicizumab is an…
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