In addition to its clinical challenges, hemophilia is characterized by a major economic burden that affects patients, care givers, the healthcare system and society. In a recent report, Sheh-Li Chen, PharmD, from the University of North Carolina Medical Center, identifies the most significant areas of healthcare hemophilia management, highlighting the importance of the…

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…

Genentech recently presented positive Phase 1/2 extension results for its investigational hemophilia A medicine, emicizumab, at the 2016 World Federation of Hemophilia (WFH) World Congress in Orlando, Florida. Emicizumab is being developed by Genentech (a Roche company) and Chugai Pharmaceutical. The latest data analysis continued to show a promising profile…

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…

uniQure N.V. has updated the clinical data from its ongoing clinical trial of a gene therapy for the treatment of patients with hemophilia B. The results indicate sustained improvement by all patients at a low-dose, with durable levels of  Factor IX (FIX) gene activity for several weeks after treatment. The…

Bayer announced that six hemophilia clinicians and researchers in the United States were among 16 grant recipients under the company’s Hemophilia Awards Program for 2016. The announcement was part of the World Federation of Hemophilia 2016 World Congress now taking place in Orlando, Florida. Grant amounts for the six were not given,…

New Phase 3 trial results of CSL Behring’s hemophilia B therapy candidate Idelvion show promise in terms of reduction of bleeds, prolonged usage, and effectiveness at low doses. The poster detailing the data, “Long-term safety and efficacy of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients…

Key findings from clinical trials of three recombinant coagulation factors as new hemophilia treatments, presented at last year’s Congress of the International Society on Thrombosis and Haemostasis (ISTH 2015), have been published in a supplemental issue of the journal Thrombosis Research. The results were presented in a session…

The US Food and Drug Administration has granted Breakthrough Therapy Designation to biotech Spark Therapeutics and pharmacology giant Pfizer for the hemophilia B treatment SPK-9001. The designation allows expedited development and FDA review of the gene therapy currently undergoing a Phase 1/2 trial as a potential one-time therapy. The trial is recruiting patients.

CSL Behring announced that new data from studies of two approved treatments for hemophilia — Idelvion and Afstyla, both recombinant coagulation factors — will be presented at the XXXII International Congress of the World Federation of Hemophilia (WFH) 2016 next week. CSL will present six posters highlighting Idelvion, a long-acting recombinant albumin fusion…