NDAINTRAVENOUSPOWDERPriority Review
Approved
Feb 2021
Lifecycle
LOE Approaching
Competitive Pressure
15/100
Data completeness
4/8 — Partial
Priority Review
NULIBRY (fosdenopterin hydrobromide) by Sentynl Therapeutics is mocs1 gene leading to deficient mocs1a/b dependent synthesis of the intermediate substrate, cpmp. Approved for mortality in patients with molybdenum cofactor deficiency (mocd) type a. First approved in 2021.
Drug data last refreshed 16h ago · AI intelligence enriched 6d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
NULIBRY (fosdenopterin hydrobromide) is an intravenous powder that provides exogenous cPMP substrate replacement for patients with molybdenum cofactor deficiency (MoCD) Type A. It works by replenishing the molybdenum cofactor pathway, enabling sulfite oxidase activation and reducing neurotoxic sulfite accumulation. This is a life-saving therapy for a rare metabolic genetic disorder with no alternative treatment.
LOE Approaching2.5 years to LOE
Small commercial footprint with limited team due to ultra-rare indication; transition planning toward loss of exclusivity in ~2.6 years will require strategic repositioning.
Mechanism of Action
MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the…
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.