Alnylam Pharmaceuticals has suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or B, after a patient died of swelling in the brain in a Phase 2 open-label extension trial. The company will work with the regulatory agencies in reviewing the cause of the…
Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…
Bioverativ will collaborate with the Boston imaging company Invicro to use ultrasound imaging to improve the diagnosis and management of joint disease in hemophilia patients. “Through this innovative collaboration with Invicro, we hope to create meaningful progress in hemophilia care by helping people with hemophilia, their caregivers, physicians and treatment team monitor…
Shire announced it is working with MicroHealth to develop a free and secure digital monitoring mobile app for hemophilia A and B patients with antibodies, known as inhibitors, that infer with clotting factors they are using to treat their disease. MicroHealth was co-founded by Aaron Craig, who has hemophilia A,…
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 has approved new vial strengths for Octopharma’s hemophilia A therapy Nuwiq, which means patients will need fewer vials per treatment session. Octapharma USA said single-dose strengths of 2,500, 3,000 and 4,000 International Units will be available in the United States in September. The vials are already…
The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a collaboration with Pfizer, is developing  the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in hemophilia A…
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…
Recent advances in gene therapy approaches to treat hemophilia B were recently announced by uniQure. The company achieved “substantial progress in advancing our lead program in hemophilia B,” Matthew Kapusta, CEO of uniQure, said in a press release. The company reacquired the development and commercial rights to…
Three patients who received SPK-8011 — a gene therapy developed by Spark Therapeutics — for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company. The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with…
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