News

Scientists in Taiwan have created a new point-of-care device to diagnose hemophilia type A and determine a person’s blood type using just a small sample of blood. The device “is especially suitable for usage in emergency or natural disasters to provide quantitative testing in rescue and relief operations,” the…

Healthcare costs are comparable for male patients with hemophilia A who are treated with either Adynovate (rurioctocog alfa pegol) or Eloctate (efmoroctocog alfa), according to a real-world study in the U.S. “Results from this analysis may be used to inform product coverage and treatment decisions valuable for both…

SerpinPC, an experimental treatment being developed by Centessa Therapeutics, continues to be safe when used for up to nearly three years and to almost zero out the number of bleeds in adults with severe hemophilia A or B. Those findings come from a one-year extension of the…

Bleeding rates and the use of replacement therapies among men with severe or moderately severe hemophilia B remain low at least three years after a single dose of the approved gene therapy Hemgenix (etranacogene dezaparvovec). That’s according to updated data from the Phase 3 HOPE-B clinical trial…

The parents of most children with severe hemophilia A who took part in the XTEND-Kids clinical trial of Altuviiio (efanesoctocog alfa) reported in exit interviews that the once-weekly treatment led to notable improvements in mood for their sons, with some reporting a greater ability for their boys to…

The once-weekly injection therapy marstacimab is being considered for approval in both the U.S. and Europe for people with hemophilia A and hemophilia B who don’t have inhibitors. The U.S. Food and Drug Administration (FDA) is expected to decide on it in late 2024, while a decision from the European…

A partnership between ARUP Laboratories and Medicover will improve access to AAV5 DetectCDx, the former’s companion diagnostic test to assess hemophilia A patients’ eligibility to Roctavian (valoctocogene roxaparvovec-rvox) in the European Union (EU). The partnership, which allows testing to be done at one of Medicover’s…

A first patient has been dosed in a Phase 1/2a clinical trial that’s assessing the safety and preliminary efficacy of ASC618, a second-generation gene therapy for hemophilia A. The Phase1/2a study (NCT04676048), which has been in the works since 2021, aims to enroll about 12 men with severe…

Eligible patients with severe hemophilia A in Germany should now have improved access to Roctavian (valoctocogene roxaparvovec-rvox) — a gene therapy intended to reduce bleed risk in certain adults with the bleeding disorder — given a new agreement between its developer BioMarin Pharmaceutical and the German National Association…

The U.S. Food and Drug Administration (FDA) has given orphan drug and rare pediatric disease designations to Sernova’s experimental and cell-based hemophilia A treatment program using the Cell Pouch System, the company’s novel medical device. Orphan drug status is awarded to therapies aiming to treat…