Orphatec Pharmaceuticals

Orphatec Pharmaceuticals was a German clinical-stage biotechnology company focused on developing treatments for ultra-rare orphan diseases. The company is best known for developing ORP-101 (fosdenopterin), a first-in-class cyclic pyranopterin monophosphate (cPMP) replacement therapy for Molybdenum Cofactor Deficiency (MoCD) Type A. MoCD Type A is a fatal biochemical disorder that causes rapid neurological damage in newborns due to the inability to produce the molybdenum cofactor. Orphatec's assets were acquired by Origin Biosciences, a subsidiary of BridgeBio Pharma, which completed the clinical development. This work culminated in the 2021 FDA approval of Nulibry, the first treatment for MoCD Type A.