A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…
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Hemophilia A is an inherited bleeding disorder primarily affecting males, characterized by a deficiency in factor VIII, a crucial blood clotting protein. While there is no cure, it can be managed with medication and lifestyle adjustments. Mild cases may go undetected by standard clotting tests, leading to diagnoses after excessive bleeding from procedures like oral surgery. Current research focuses on improving treatments, including studies on managing hemophilia A with inhibitors and evaluating preventive therapies like emicizumab.
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Excessive bleeding after oral surgery led to two people in China, both with normal pre-operative blood clotting tests, being diagnosed with hemophilia A, according to a recent case report. Mild hemophilia can go undetected in standard laboratory tests, and oral surgeons need to be prepared for such cases, the research…
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…
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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Explaining Hemophilia A and B
https://www.youtube.com/watch?v=S_YMGmHqIW4 This video from Claire Blatt shares a lecture about the effects of two blood-clotting disorders: hemophilia A and hemophilia B. MORE: A study shows that NovoSeven counters bleeding in children with hemophilia Though the lecture is aimed at nurses, it’s helpful for anyone looking to learn about these…
The Medicines and Healthcare Products Regulatory Agency (MHRA) for the U.K. has approved the resumption of patient enrollment in BioMarin Pharmaceuticals’ Phase 1/2 clinical trial evaluating the investigational gene therapy BMN 270 as a treatment for severe hemophilia A. BioMarin suspended patient dosing in the trial in June because of increasing levels…
Clinical trials of Hemlibra (emicizumab-kxwh) continue to show significant reductions in bleeding episodes in patients with hemophilia A with inhibitors, Genentech recently announced. Data from Hemlibra’s clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. The results show that in hemophilia A patients with inhibitors…
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 U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the U.S. into the safety and early efficacy of ASC618, its second-generation gene therapy for hemophilia A. A Phase 1/2 trial (NCT04676048), set to start this month, will test…
Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…