fosdenopterin

Fosdenopterin is a first-in-class synthetic form of cyclic pyranopterin monophosphate (cPMP) used as a substrate replacement therapy for molybdenum cofactor deficiency type A (MoCD-A), a rare and life-threatening genetic disorder. Patients with MoCD-A have mutations in the MOCS1 gene, resulting in an inability to synthesize cPMP, which is essential for the biosynthesis of the molybdenum cofactor (MoCo). MoCo is required for the activity of several enzymes, including sulfite oxidase, xanthine dehydrogenase/oxidase, and aldehyde oxidase. Deficiency leads to accumulation of toxic sulfites and S-sulfocysteine in the central nervous system, causing severe neurological damage and early mortality. Fosdenopterin provides exogenous cPMP to restore MoCo biosynthesis and enzyme function[2][5][6][8]. It was developed at TU Braunschweig and University of Cologne[5] and approved by FDA under priority review for reducing mortality risk in patients with MoCD-A[5].

Brand names

Nulibry

Other names

cyclic pyranopterin monophosphatecPMP