Steve Bryson, PhD,  science writer—

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…

Adults with hemophilia and ankle joint damage taking part in a yearlong pilot study largely reported that two injections of hyaluronic acid, which acts to lubricate the joint, eased ankle pain and improved movement. The treatment, given six months apart in this U.K. trial, also enabled most of these people…

Immune checkpoint inhibitors (ICIs) used for cancer treatment may be associated with an increased likelihood of developing acquired hemophilia A, according to an analysis of an adverse events database. Although the small number of reports do not confirm that these therapies are causing the bleeding disorder, early identification and…

Hepatitis C virus (HCV) infection remains a major risk factor for hepatocellular carcinoma — the most common type of liver cancer — in men with hemophilia, despite the wide availability of medications to fight the virus, a U.S. database analysis has found. Other independent risk factors for the cancer…

People in France with severe hemophilia A who switched to Elocta (efmoroctocog alfa) — an extended half-life replacement therapy — had fewer infusions and a reduced rate of joint bleeds, a real-world study reports. Notably, those who switched to Elocta had a higher bleeding rate, including joint bleeds,…

One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed. Reduced pain intensity in the elbows also…

The gene therapy BBM-H901 increased factor IX activity in 10 men with moderate to severe hemophilia B, while reducing bleeds and the use of FIX replacement therapy over one year, according to results of a pilot Phase 1 trial. “The study was the first clinical trial conducted by Belief BioMed…

The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for EtranaDez (etranacogene dezaparvovec), an investigational gene therapy for adults with hemophilia B. The FDA grants priority review to investigational therapies designed to treat serious medical conditions. If approved, the treatment…