Margarida Azevedo, MSc,  —

CSL Behring marked World Hemophilia Day April 17 by shipping more than 10 million international units (IUs) of its medicines to treat hemophilia A to developing countries through the nonprofit World Federation of Hemophilia (WFH). The medicines were sent to countries like Vietnam, Cameroon and others through the WFH’s…

The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…

The five adults with hemophilia A who died while using Hemlibra (emicizumab-kxwh) to treat their disease did not die of causes related to the treatment, manufacturer Genentech said in a statement. “In each of these cases, the assessment of the treating physician or investigator was that the cause of death was unrelated to…

BioMarin Pharmaceutical has won an award for getting its new gene therapy facility in Novato, California, operational ahead of schedule. The International Society for Pharmaceutical Engineering (ISPE) award was for project execution. It was part of the society’s 2018 Facility of the Year honors. BioMarin took only a year from the time…

Patients with hemophilia B are reporting a mostly positive response to treatment with Ixinity (recombinant coagulation factor IX), according to new data from Aptevo Therapeutics, the therapy’s maker. The company presented the patient-reported outcomes data at the recent Thrombosis and Hemostasis 2018 Summit of North America in San Diego. The event is…

Sigilon Therapeutics’s Afibromer SIG-003 candidate for the long-term treatment of patients with hemophilia B recently received an Advanced Therapy Medicinal Product (ATMP) designation in the European Union. The designation was granted by the Committee for Advanced Therapies of…

The National Hemophilia Foundation (NHF) kicked off its 70th anniversary with an advocacy effort on Capitol Hill in Washington, D.C. At the same time, the NHF launched its 2018 Red Tie Campaign  to raise awareness about Bleeding Disorders Awareness Month and reinforces its campaign to oppose the U.S. Congress’ efforts to terminate…

myPKFiT for Advate, a free web-based software designed to personalize and assess preventive dosing regimens for some hemophilia A patients treated with Advate, is now available in the U.S. MyPKFiT for Advate is the first and only pharmacokinetic (PK) dosing software approved by the U.S. Food and Drug Administration (FDA)…

The European Commission has approved Hemlibra (emicizumab) for routine preventive treatment of bleeding episodes in people who have hemophilia A with factor VIII inhibitors. The antibody therapy is designed to restore the factor VIII, which is deficient in hemophilia A. Hemlibra is being co-developed by Japan’s Chugai Pharmaceuticals, Switzerland’s…

AGC Biologics has agreed to produce the hemophilia B clotting therapy that Catalyst Biosciences is testing in clinical trials, the companies announced. The next step in testing CB 2679d will be a Phase 2b trial in the third quarter of 2018, according to Catalyst, which is based in South San…