Nov 15, 2024 · Centessa Pharmaceuticals is discontinuing the clinical development of SerpinPC, its experimental under-the-skin therapy for people with hemophilia A and hemophilia B with or without inhibitors. The therapy was being evaluated in several Phase 2 clinical trials.

Nov 29, 2024 · Adults and adolescents with hemophilia A or B without inhibitors saw significantly fewer bleeding episodes with concizumab as prophylaxis.

Nov 18, 2024 · There are many support groups and resources available to help people affected by hemophilia A manage and cope with the bleeding disorder.

Oct 25, 2024 · Hympavzi (marstacimab-hncq) may be available by prescription in the U.S. before the end of the year. The therapy was approved this month as a routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with hemophilia A or B who haven’t developed inhibitors.

Oct 11, 2024 · The FDA has approved Pfizer’s marstacimab under the brand name Hympavzi for adults and children with hemophilia A or B, without inhibitors.

Oct 15, 2024 · Spanish authorities issued a positive recommendation for national reimbursement of Hemgenix gene therapy for eligible hemophilia B patients.

Dec 17, 2024 · Preventive treatment with Altuviiio led to lower bleed rates and better joint health than Hemlibra in hemophilia A patients in an analysis.

Nov 19, 2024 · Metagenomi, which is developing MGX-001, a gene-editing therapy for hemophilia A, is preparing to initiate investigational new drug (IND)-enabling activities that seek to support the therapy’s transition from preclinical to clinical testing.

Feb 7, 2024 · In over seven years of follow-up, a single dose of Roctavian reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%.

Sep 27, 2023 · Hemgenix, formerly known as AMT-061 and EtranaDez, is a one-time gene therapy used to treat bleeding episodes in hemophilia B patients.