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.
Rebecca Voelker. New Option for Bleeding Disorder. JAMA. 2016;315(3):243. doi:10.1001/jama.2015.18731