CAR T-cell therapy, a type of treatment that weaponizes the body’s immune system and was originally developed to treat cancer, for the first time successfully treated a patient with acquired hemophilia A (AHA), a study reported. The study, “Anti-CD19 CAR-T cell therapy for acquired hemophilia A,”…

The blood-clotting protein factor VIII (FVIII) that’s missing or faulty in people with hemophilia A is flexible and adopts multiple shapes, some of which favor the binding of neutralizing antibodies that can reduce the effectiveness of FVIII replacement therapies, researchers have learned. “This helps explain why some people…

Jivi (damoctocog alfa pegol) has received approval from the U.S. Food and Drug Administration (FDA) that now extends to children with hemophilia A as young as 7 years old. Jivi had previously been authorized for hemophilia A patients 12 and older. The therapy, which is sold by Bayer,…

In 2000, actor Tom Hanks starred in the movie “Cast Away.” His character, Chuck Noland, traveled the world as a systems analyst for FedEx, and his plane crashed in the South Pacific. He was on a deserted island, and his only connections with the world were the FedEx…

The National Institute for Health and Care Excellence (NICE) has issued a final guidance recommending the use of Altuvoct (efanesoctocog alfa) in England and Wales as an option for people with severe hemophilia A, ages 2 and older. The National Health Service, the U.K.’s public healthcare system, is…

Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery. “This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie…

The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older, with or without inhibitors, or antibodies that can make treatment less effective.

Takeda Pharmaceuticals has decided to discontinue Hemofil M (human antihemophilic factor) and Recombinate (recombinant antihemophilic factor), both of which are approved as replacement therapies to prevent and control bleeding episodes in children and adults with hemophilia A. “This was not a decision we made lightly,” Anthea Cherednichenko,…

Healthcare costs, particularly related to disease-specific medications, are especially high for people with hemophilia A or hemophilia B on preventive treatments and/or for those with inhibitors. That’s according to a study in France, which also found that patients have a significant clinical burden, with a higher risk of…