Steve Bryson, PhD,  science writer—

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…

A significant proportion of bleeds in people with hemophilia A are left untreated, according to an analysis of data from an observational study. Because only treated bleeds are typically reported in clinical trials, these findings suggested the full disease burden is not adequately captured in many studies. As such,…

If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient compared with other preventive treatments, specifically Hemlibra. That’s according to a draft evidence report issued by pricing watchdog the Institute for Clinical and Economic Review (ICER). An independent non-profit…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SerpinPC for treating hemophilia B. Orphan drug status is awarded to therapeutics intended to treat rare conditions, defined as those affecting fewer than 200,000 people in the U.S. The designation provides financial incentives to support clinical…

A participant in a Phase 3 clinical trial evaluating Roctavian (valoctocogene roxaparvovec), a gene therapy for hemophilia A, has been diagnosed with leukemia. A genetic assessment of the case, conducted by the therapy’s developer, BioMarin Pharmaceutical, suggested the cancer diagnosis was not linked to Roctavian. Details of…