FDA grants regulatory approval of fosdenopterin (Nulibry) for molybdenum cofactor deficiency (MoCD) Type A

Approval of this first-in-class cyclic pyranopterin monophosphate substrate replacement therapy, was based on studies which demonstrated reduced mortality vs untreated, genotype-matched, historical controls (HR=0.18, 95% CI 0.04, 0.72).

Source:

Biospace Inc.