On December 23rd, the BioMarin submitted a Biologics License Application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration.
Sangamo transferred the investigational new drug application for their hemophilia A gene therapy candidate to Pfizer, which will now lead its phase 3 clinical development.
Roche is poised to aquire Spark Therapeutics and it's hemophilia A gene therapy portfolio for $4.3 billion.
The FDA first approved Jivi® in August 2018 for the treatment of hemophilia A in adolescents and adults 12 years of age and over, including for prophylactic, on-demand and perioperative management of bleeding.
Pfizer and Sangamo presented their gene therapy updates in a poster presentation during the 61st Annual Meeting of the American Society of Hematology in Orlando, Florida.
The therapy is for patients with severe and moderately severe hemophilia B.
NHF and HFA have submitted another letter to Bayer requesting information on this serious event.
UniQure plans to exceed its original recruitment goal by "over-enrolling" an additional six trial participants by the end of September.
Marstacimab would represent an alternative approach to standard factor VII or factor IX replacement therapy.
Orphan Drug Designation is awarded by the U.S. FDA to therapies designed to treat rare diseases affecting fewer than 200,000 people in the United States.
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