The U.S. Food and Drug Administration (FDA) has cleared for use CRYOcheck Chromogenic Factor VIII, Precision BioLogic‘s lab test to monitor factor VIII activity in the blood of hemophilia A patients ages 2 and older and help in disease management, the company said in a…
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New developments in hemophilia A include the FDA clearance of CRYOcheck for monitoring factor VIII activity. The MOTIVATE study is collecting real-world data on managing hemophilia A with inhibitors. Access to Elocta is expanding in the UK. Roctavian, a gene therapy, is awaiting approval from the EMA and FDA. Hemlibra is considered comparable or superior to FVIII replacement therapy.
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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…
People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog alfa) under a two-year agreement between Sobi and the National Health Service (NHS), the company announced. Elocta was approved by the European Commission for the treatment and prevention of bleeding in all…
The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…
At standard U.S. doses, Hemlibra (emicizumab) provides comparable or even superior clinical benefits than prophylactic factor VIII (FVIII) replacement therapy among adults with severe hemophilia A and no inhibitors, according to a report from the Institute for Clinical and Economic Review (ICER). Hemlibra is also cost-saving, due to…
Novo Nordisk has resumed its Phase 3 clinical trials investigating concizumab as a potential treatment for hemophilia A and B, the company announced in a press release. The trials were paused in March due to the occurrence of non-fatal thrombotic (blood-clotting) events in three patients. In collaboration…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment of episodic bleeds in hemophilia A and B patients with inhibitors. MarzAA, developed by Catalyst Biosciences, will…
ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program. “We are proud to welcome Vigene, a global leader in gene therapy process development and GMP [Good Manufacturing…
Although hemophilia treatments are generally effective, women and girls with this disease have specific medical challenges, such as excessive menstrual bleeding, that must be addressed for better clinical care. A study evaluated the symptoms, bleeding management, and clinical outcomes related to menstruation, childbirth, dental procedures, surgeries, and other bleeding events among female…
Immunoadsorption, or the selective removal of antibodies from the blood, might prove useful as an add-on treatment for patients with acquired hemophilia, according to a recent study. If confirmed, this finding could lead to better treatment for life-threatening bleeds, delayed treatment responses, and adverse responses to immunosuppressive therapy among…