Roche's heavy-duty drug for type A haemophilia, Hemlibra, gets FDA approval

type A haemophilia is a serious genetic diseasehealthy(when a person bleeds, a clotting protein called factor VIII brings together factor IXa and factor X, triggering a clotting process that helps stop the bleeding)recently, the Roche Group announced that itthe FDA's(emicizumab-kxwh), a heavy-duty drug that approves its treatment of type A haemophilia, as a routine preventive therapy for patients with type A haemophiliacs who do not contain factor VIII inhibitors, to prevent or reduce frequent bleedingHemlibraHemlibra is currently the only type A haemophiliac with or without factor VIII inhibitors in the body, and allows patients to receive subcutaneous injections weekly, biweekly or every four weeksHemlibra is a bispecific factor IXa and factor X antibody that brings together the factors IXa and Factor X needed to activate the natural coagulation cascade to help patients with type A haemophiliacs restore clotting functionHemlibra is a preventive subcutaneous injectable therapy, discovered by Roche's JapanesePharmaceuticaland developed by Chugai, Roche and GenentechHemlibra was approved in November last year for routine preventive therapy for adults and children with type A haemophiliacs containing factor VIII inhibitors in the bodyThe drug also obtained a breakthrough treatment and priority review for type A haemophilia, which does not contain factor VIII inhibitors in the bodyrelated research
This approval is based on the results of the 3-phasetest (3 and HAVEN 4) In phase 3 of trial HAVEN 3, a total of 71 adolescents over 12 years of age and adults with type A haemophilia were divided into two groups, one group receiving Hemlibra prophylaxis once a week (n-36) and the other receiving treatment every two weeks (n-35) The bleeding conditions in both groups requiring treatment were reduced by 96% (95% CI: 92.5; 98.0, p 0.0001) and 97% (95% CI: 93.4; 98.3, p 0.0001), and compared to the non-preventive treatment group (n?18) at the various endpoints associated with bleeding, the safety was also good in addition, Hemlibra in a trial of patients who had previously received the traditional standard therapy for Factor VIII (n-48), achieved a 68% reduction in bleeding conditions (95% CI: 48.6; 80.5, p 00.00) In the single arm Phase 3 trial HAVEN 4, a total of 41 patients with type A haemophiliacs received preventive treatment every four weeks, of which good clinically significant bleeding control results were shown in patients with (n?5) and no (n-36) factor VIII inhibitors, and the safety was good