Steve Bryson, PhD, science writer —

Venture philanthropy fund Pathway to Cures (P2C)) has invested $500,000 in Five Liters, which is developing noninvasive, nonpharmacological neuromodulation therapies for treating inheritable blood and bleeding disorders, such as hemophilia and von Willebrand disease (VWD). “Investing in Five Liters’ early-stage research is a critical step in addressing unmet…

The protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on its own to stimulate immune responses in a mouse model, a study suggests. This immune response to FVIII is dependent on a set of distinct immune cells within the spleen,…

Novo Nordisk’s Alhemo (concizumab injection) has been approved in Canada for hemophilia A patients, 12 years or older, who test positive for factor VIII inhibitors. The under-the-skin injectable therapy was recently approved by Health Canada for hemophilia B patients in the same age group with factor…

Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in adults with mild or moderate hemophilia A without inhibitors, according to a modeling study. The cost of Hemlibra would need to drop by at least 35% to become cost-effective…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to SerpinPC, Centessa Pharmaceuticals’ investigational treatment for people with hemophilia B. Fast track status expedites the review of therapy candidates that show a potential to fill an unmet medical need for serious conditions. It also allows more…

Hemgenix (etranacogene dezaparvovec), the first gene therapy approved for adults with hemophilia B, has been shown to work better than standard preventive replacement therapy to lower the frequency of bleeds — both spontaneous and joint bleeds — in this patient population. The therapy also was superior in…

The European Commission has conditionally approved Hemgenix (etranacogene dezaparvovec), a one-time gene therapy to treat adults with hemophilia B. Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. It’s the first gene therapy for…

The European Commission has approved Hemlibra (emicizumab) as a routine preventive treatment for people with moderate hemophilia A without inhibitors. “We welcome the European Commission’s decision to approve Hemlibra also for people with moderate hemophilia A in the EU,” said Levi Garraway, MD, PhD, Roche’s chief medical officer…

The U.S. Food and Drug Administration (FDA) has cleared CRYOcheck Chromogenic Factor IX assay, a test intended to be used by clinical labs to detect a deficiency in factor IX (FIX), the cause of hemophilia B. The chromogenic, or color-based, test developed by Precision BioLogic is reported…

A review is continuing into a resubmitted application for approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with severe hemophilia A, with a planned advisory committee meeting having been canceled. The U.S. Food and Drug Administration (FDA) had requested, but now no longer intends, to meet with…