Partners Sanofi SA and Swedish Orphan Biovitrum AB (Sobi) said regulatory submissions are expected this year for once-weekly factor VIII therapy efanesoctocog alfa in hemophilia A following top-line success in a pivotal phase III study, which showed a clinically meaningful prevention of bleeds in people with severe disease receiving prophylaxis over 52 weeks. The drug, also known as BIVV-001, has fast track and orphan designations in the U.S., and the companies are banking on its extended half-life to go up against blockbuster bispecific antibody Hemlibra (emicizumab) from Roche Holding AG as well as a potential gene therapy from Biomarin Pharmaceutical Inc.
LONDON – Targed Biopharmaceuticals BV has raised €39 million (US$44.2 million) in a series A financing that will enable it to take its targeted clot busting drug Microlyse into clinical development. The first-in-class product consists of urokinase, a serine protease involved in the conversion of inactive plasminogen to active plasmin, linked to a nanobody targeted at von Willebrand factor, the blood glycoprotein that plays a key role in hemostasis.
The FDA’s approval of Agios Therapeutics Inc.’s Pyrukynd (mitapivat) for treating hemolytic anemia marks a turnaround from nearly a year ago. That’s when Agios sold its commercial, clinical and research-stage oncology portfolio to privately held Servier Pharmaceuticals LLC to concentrate on rare diseases.
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