News

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…

FDA Grants Breakthrough Designation for Hemophilia B Treatment

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…

Biogen and Sobi to Present Long-term Data on Hemophilia A and B Therapies at WFH 2016

Biogen and Sobi  announced that they will be presenting updated and long-term data on two approved hemophilia treatments, Eloctate  (marketed as Elocta in Europe) for hemophilia A, and Alprolix for hemophilia B, at the World Federation of Hemophilia (WFH)’s 2016 World Congress next week. Specifically, data from separate Phase 3 extension studies evaluating each treatment’s safety and…

CSL to Present New Data on 2 Hemophilia Treatments, Idelvion and Afstyla, at the WFH Congress

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…

Advantages, Unanswered Questions About PEGylated Products for Hemophilia A Reviewed by Researchers

Researchers reviewed the current hemophilia research that focuses on the development of improved longer-acting factor replacement therapies, particularly PEGylated products. The new clinical research advances lead the scientific community to look at such developments with optimism, but also some caution. The study, “Potential role of a new PEGylated recombinant…