BioMarin Investigational Gene Therapy Updates

DNA strand

An update for the hemophilia community from BioMarin regarding a previously reported serious adverse event in the ongoing hemophilia A gene therapy [valoctocogene roxaparvovec, BMN 270] Phase 3 study that was deemed, by the Independent Data Safety Monitoring Committee and the company, very unlikely to be related to the investigational therapy. Clinical Development Program Update […]

STAQ Pharma, Inc. Advises of Printing Error

STAQ Pharma, Inc. has advised the Hemophilia Alliance and the National Hemophilia Foundation of a typographical printing error on a few units of lot 21114255A Desmopressin Acetate 1.5 mg/mL. This does not involve any patient safety issues. The expiration date on a few of the vials shows 2021-06-28 (June 28, 2021) when the correct expiration […]

FDA Accepts CSL Behring’s Biologics License Application for Etranacogene Dezaparvovec for Priority Review  

Global biotherapeutics leader CSL Behring, a business of CSL, announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA), for priority review, for etranacogene dezaparvovec (also known as CSL222), an investigational gene therapy for the treatment of adults with hemophilia B. Etranacogene dezaparvovec was specifically designed to make near-normal […]

FDA Grants Breakthrough Therapy Designation for Hemophilia A

HFA received the following press release from Sanofi. FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA Designation is based on XTEND-1 Phase 3 study data demonstrating a clinically meaningful prevention of bleeds and superiority in prevention […]

Bayer Discontinues Kogenate® FS in the United States

HFA received the following update from Bayer. For more than 25 years, Bayer has remained committed to supporting patients around the world by keeping a constant pulse on their evolving needs as well as those of their caregivers and healthcare providers (HCPs). Recognizing the growing shift in patient use toward more recent products, such as […]