BioMarin Update: FDA Extends PDUFA Target Action Date to June 30, 2023
SAN RAFAEL, Calif., March 6, 2023 — BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company’s Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for adults […]
FDA Approves Sanofi’s Once-Weekly ALTUVIIIO™
Source: Sanofi Press Release Sanofi announces that the U.S. Food and Drug Administration approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-vWF-XTEN Fusion Protein-ehtl], previously known as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A. Sanofi states that with ALTUVIIIO, they are looking to raise the bar for people with […]
BioMarin Investigational Gene Therapy Updates
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 […]
Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment
FDA Grants Priority Review to Efanesoctocog Alfa for People with Hemophilia A
Fitusiran prophylaxis reduced bleeds by 61% in people with Hem A or B
Once-Weekly Efanesoctocog Alfa: an Update from Sanofi
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 […]