Preliminary results of a Phase 1/2a trial of the hemophilia A experimental treatment BIVV001 show a significant extension of replacement therapy lifetime in the blood, Bioverativ announced. The ongoing, and still recruiting, open-label, multicenter EXTEN-A study (NCT03205163) is evaluating the safety, tolerability, and pharmacokinetics of a single intravenous injection…

Should regulatory authorities agree with favorable results from Genentech’s ongoing Phase 3 studies of Hemlibra (emicizumab-kxwh),  hemophilia A patients may have a treatment they can use regardless of inhibitor status, with a choice of three dosing schedules that might better match their lifestyle and preferences. “The ultimate goal for…

Data from ongoing Phase 3 clinical trials show that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis — or preventive treatment — greatly outperforms current standard-of-care therapies, leading to significant reductions in the number of bleeds in hemophilia A patients regardless of inhibitor status. Promising results from these pivotal studies may lead…

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…

Researchers are creating a free web-based calculator that will help physicians determine the best patient-specific preventive regimens for children with hemophilia, according to a recent study. The tool is based on newly developed methods that identified key features of an effective preventive regimen. This includes giving Factor VIII (FVIII) injections…

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…

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…

Eloctate use in immune tolerance induction (ITI) therapy shows promise in high-risk patients with severe hemophilia A and inhibitors treated for the first time, Bioverativ announced. The results support a potential benefit for some patients who tried and failed ITI with other factors. The study, “Recombinant factor VIII…

Shire Korea recently received approval from South Korea’s Ministry of Food and Drug Safety for Adynovate (BAX 855), a long-lasting factor VIII gene recombinant therapy for hemophilia A. Adynovate is an injectable form of Shire’s Advate, which is Korea’s most commonly used treatment for the blood disorder. Shire Korea…

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…