Regulatory news: Nulibry (fosdenopterin) approved to reduce the risk of mortality in patients with molybdenum cofactor deficiency type A: FDA approval summary

J Inherit Metab Dis. 2021 Sep;44(5):1085-1087. doi: 10.1002/jimd.12421. Epub 2021 Aug 8.

Authors

Sheila Farrell¹, Jacqueline Karp¹, Rebecca Hager², Yan Wang², Oluseyi Adeniyi³, Jie Wang³, Liang Li³, Lian Ma³, Jackye Peretz¹, Mukesh Summan¹, Nicolas Kong¹, Michael White¹, Michael Pacanowski³, Dionne Price², Jane Filie¹, Kathleen Donohue¹, Hylton Joffe¹

Affiliations

¹ Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
² Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
³ Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.

PMID: 34337775
DOI: 10.1002/jimd.12421

No abstract available

Publication types

Editorial
Review

MeSH terms

Animals
Clinical Studies as Topic
Humans
Kaplan-Meier Estimate
Metal Metabolism, Inborn Errors / mortality
Molybdoferredoxin / drug effects
Organophosphorus Compounds / adverse effects
Organophosphorus Compounds / pharmacology
Organophosphorus Compounds / therapeutic use*
Pterins / adverse effects
Pterins / pharmacology
Pterins / therapeutic use*
United States
United States Food and Drug Administration

Substances

Molybdoferredoxin
Organophosphorus Compounds
Pterins
nulibry

Supplementary concepts

Molybdenum Cofactor Deficiency, Complementation Group A