Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.
Search results for:
A single dose of BIVV001 significantly increased levels of factor VIII for a week in the blood of people with severe hemophilia A, according to early results of a Phase 1/2a study. The treatment increased the half-life of factor VIII to 44 hours and showed no side effects or development…
A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia A. These preliminary findings from an ongoing Phase 1/2 trial (NCT03734588) were presented by Spencer Sullivan, MD,…
The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as a…
Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about a year of treatment. That’s according to top-line data from the Phase 3 XTEND-Kids trial (NCT04759131) announced by Sobi and Sanofi, the therapy’s co-developers, less than a month after…
Dr. Reddy’s Laboratories announced the U.S. launch of a generic version of DDAVP (desmopressin acetate), used to control bleeding in people with mild hemophilia A. The generic is available under the name Desmopressin Acetate Injection USP, 4 micrograms/mL, in a carton of 10 single-dose ampules…
The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog beta for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors. A…
A new five-year alliance between Bayer and the World Federation of Hemophilia (WFH) Humanitarian Aid Program will deliver training, education and treatments to healthcare professionals in more than 60 underserved countries. ”Three out of four people with bleeding disorders living in developing countries do not have access to…
Jivi (damoctocog alfa pegol) is an approved lab-made replacement therapy designed to prevent or treat bleeding episodes in previously treated children and adults with hemophilia A. It is administered via into-the-vein (intravenous) infusions.
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…