The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29. ICER’s report will be subject to a public deliberation in March 2018 by the…

Dimension Therapeutics may soon become a wholly owned subsidiary of Regenxbio, following the terms of an agreement between the two companies. The merger agreement, if finalized, will add Dimension’s investigational drug candidates being developed to Regenxbio’s pipeline, including DTX201 for the treatment of hemophilia A. DTX201 was being developed…

Alnylam Pharmaceuticals has suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or B, after a patient died of swelling in the brain in a Phase 2 open-label extension trial. The company will work with the regulatory agencies in reviewing the cause of the…

Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…

The U.S. Food and Drug Administration has approved new vial strengths for Octopharma’s hemophilia A therapy Nuwiq, which means patients will need fewer vials per treatment session. Octapharma USA said single-dose strengths of 2,500, 3,000 and 4,000 International Units will be available in the United States in September. The vials are already…

The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a collaboration with Pfizer, is developing  the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in hemophilia A…

The U.S. Food and Drug Administration (FDA) has granted priority review to the therapy emicizumab for patients with hemophilia A with factor VIII inhibitors, Genetech, the drug’s developer, announced. The FDA also announced the acceptance of Genetech’s Biologics License Application (BLA) for emicizumab prophylaxis (preventive) therapy in adults, adolescents and children…

https://www.youtube.com/watch?v=pTIVORH7anc This video from Wochit News is all about a new treatment for severe hemophilia A. The Children’s Hospital of Los Angeles, California, has developed a breakthrough treatment that can reduce the bleeding episodes in people living with hemophilia A by up to 90 percent. MORE: Emicizumab shows…

Three patients who received SPK-8011 — a gene therapy developed by Spark Therapeutics — for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company. The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with…

BioMarin Pharmaceutical  will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 for hemophilia A. BioMarin will conduct two separate trials testing the effectiveness and safety of two different doses of BMN 270. Both trials are expected to initiate in the…