News

EMA Weighs Easing Clinical Trial Demands to Speed Testing of Hemophilia Therapies

The European Medicines Agency (EMA) is considering lowering the minimum number of patients required in clinical trials of potential hemophilia treatments because of “dramatic” increases in hemophilia research and concerns about the availability of patients for rare diseases like hemophilia A. “EMA began the process after becoming concerned that the minimum…

New Model Might Improve Hemophilia Factor VIII Treatment After Surgery

Researchers have created a model for how factor VIII replacement concentrates behave in the body of hemophilia A patients undergoing surgery, which could allow doctors to calculate more accurate and individualized doses for factor replacement treatment. The study “A Population Pharmacokinetic Model For Perioperative Dosing Of Factor VIII In Hemophilia A…

FDA Approves Easier Preparation System for Hemophilia A Patients on Adynovate Therapy

Shire recently announced that the U.S. Food and Drug Administration (FDA) has approved its Baxject III reconstitution system for Adynovate, an approved treatment for Hemophilia A. This system will allow patients and caregivers to prepare the therapy with fewer steps, minimizing the chances for possible errors. Adynovate is a recombinant Factor VIII (rFVIII) treatment with…

Gene Therapy for Severe Hemophilia A, BMN 270, Showing Early Promise in Clinical Trial

BioMarin Pharmaceuticals presented positive interim data from an open-label Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for severe hemophilia A. The promising results, presented at the XXXII International Congress of the World Federation of Hemophilia (WFH), showed that patients had improved and sustained clotting function. BMN…