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News From the Food and Drug Administration
January 19, 2016

New Option for Bleeding Disorder

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Copyright 2016 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA. 2016;315(3):243. doi:10.1001/jama.2015.18731

The first recombinant product used to treat adults with the congenital bleeding disorder von Willebrand disease has received FDA approval.

Vonicog alfa, marketed as Vonvendi, is produced with a genetically engineered Chinese hamster ovary cell line that expresses von Willebrand factor and the blood-clotting protein factor VIII. Even though von Willebrand disease affects about 1% of the US population, the FDA granted vonicog alfa orphan drug status, which usually is reserved for drugs intended to treat rare diseases.

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