A branch of the European Medicines Agency (EMA) has recommended that the experimental therapy denecimig be approved to treat adults and children with hemophilia A, with or without inhibitors. The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will be reviewed by the European…
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SerpinPC, an experimental hemophilia therapy, at high dose safely reduced overall bleed rates by up to 88% and spontaneous joint bleeds by up to 94% in adults with severe hemophilia A and B, who were not using preventive treatment, according to top-line results from a Phase 2a…
Since hemophilia was discovered, it has been accepted as a male disease. No one questions that a man is struggling with hemophilia. A man with a bleeding disorder living in a developed country is rarely denied access to healthcare. When he receives a diagnosis, he is told that…
Albumin fusion is a molecular technology that has been used to create replacement therapies for hemophilia with an extended half-life — treatments that are more long-lasting in the body and can therefore be administered less frequently.
Starting factor VIII (FVIII) preventive therapy before the age of 2.5 years improves joint outcomes in young adults with hemophilia A compared to starting treatment later in childhood, a clinical trial shows. The study, “ Young adult outcomes of childhood prophylaxis for severe hemophilia A:…
Shire and Rani Therapeutics have partnered to investigate the use of oral Rani Pill technology as the carrier system for clotting factor VIII in hemophilia A patients. As part of Shire’s commitment, the Irish pharmaceutical company made an equity investment in Rani. Under the agreement — whose terms weren’t…
Xyntha (moroctocog alfa) is a recombinant clotting factor replacement therapy approved to prevent and treat bleeds, including those occurring during surgery, in people with hemophilia A.
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…
Note: This story was updated July 30, 2024, to clarify patients participating in AFFINE had moderately severe to severe disease, and that a secondary study goal involved the mean treated annualized bleeding rate. Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well…
Once-weekly infusions of Altuvoct (efanesoctocog alfa) — a long-lasting hemophilia treatment marketed in the U.S. as Altuviiio — reduced bleeding in adults and children with hemophilia A for whom preventive treatment was not sufficiently effective. That’s the key finding of a new French real-world study that tracked…