Steve Bryson, PhD,  science writer—

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…

Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then move in pivotal clinical trials of SerpinPC — an investigational therapy for hemophilia A and B patients, regardless of disease severity or inhibitor status — that it plans to launch…

Health Canada has expanded its approval of Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes in hemophilia B to patients under age 18. The therapy, developed and marketed by Novo Nordisk, is now available to all hemophilia B patients as a preventive (prophylactic)…

BioMarin Pharmaceutical has resubmitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Roctavian (valoctocogene roxaparvovec), potentially the first gene therapy for adults with severe hemophilia A. The FDA delayed the company’s initial approval request in 2019, with the agency requiring two more…