The hemophilia A treatment Nuwiq (simoctocog alfa) is safe and effective for patients with severe disease, both when used on demand or as a preventative treatment, researchers determined in a recent report. The researchers, who analyzed available data on Nuwuq, detailed their finding in the article, “Efficacy and…
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The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29. ICER’s report will be subject to a public deliberation in March 2018 by the…
Using Hemlibra (emicizumab) as prophylaxis lowers treatment costs considerably for people with hemophilia A compared to preventative factor (FVIII) treatment, according to an economic model study. The model predicts $47,814 in savings to U.S. payers in year one, and a potential for more $3 billion in savings over 20 years…
FLT180a, also known as verbrinacogene setparvovec, is an investigational gene therapy that was investigated as a treatment for people with hemophilia B.
Hemophilia A is a rare inherited bleeding disorder and the most common type of hemophilia, accounting for about 80% of cases. It is caused by mutations in the F8 gene that lead to a deficiency in a blood-clotting protein called factor VIII (FVIII). The lack of FVIII…
Chugai Pharmaceutical filed a New Drug Application for the use of emicizumab as a potential treatment to prevent or decrease the occurrence of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. The application is based on…
Shire has asked the U.S. Food and Drug Administration to grant Investigational New Drug status to its hemophilia A treatment SHP654. The gene therapy helps generate a clotting protein known as factor VIII that is missing or defective in hemophilia A. Shire said an FDA approval of its request will…
Upfront treatment with Hemlibra (emicizumab-KXWH) may help reduce hospitalization costs associated with treating bleeds in older adults with acquired hemophilia A (AHA), according to a study. While Hemlibra itself carries a hefty price tag, economic models indicate that using the therapy may help shorten hospital stays and reduce…
Patients with hemophilia A who switched to Kovaltry (octocog alfa) — a recombinant, or lab-made, factor VIII (rFVIII) replacement therapy given at home — had significantly fewer bleeds than with their previous standard half-life replacement therapies, a real-world study in Italy reported. The therapy also reduced the frequency…
Antibody therapy Mim8 (denecimig) was well tolerated and provided effective bleed control in children with hemophilia A regardless of inhibitor status. That’s according to interim data from the now-complete Phase 3 FRONTIER3 study (NCT05306418), which enrolled children ages 1-11. These findings were consistent with recent data from…