Fosdenopterin

Fosdenopterin
Clinical data
Trade names	Nulibry
Other names	Precursor Z, ALXN1101
License data	US DailyMed: Fosdenopterin;
Routes of; administration	Intravenous
ATC code	A16AX19 (WHO) ;
Legal status
Legal status	US: ℞-only; EU: Rx-only;
Identifiers
	IUPAC name 5-amino-11,11,14-trihydroxy-14-oxo-13,15,18-trioxa-2,4,6,9-tetraza-14λ5-phosphatetracyclo[8.8.0.03,8.012,17]octadeca-3(8),4-dien-7-one;
CAS Number	150829-29-1;
PubChem CID	135894389;
DrugBank	DB16628;
ChemSpider	17221217;
UNII	4X7K2681Y7;
KEGG	D11779;
ChEMBL	ChEMBL2338675;
CompTox Dashboard (EPA)	DTXSID90934067 ;
Chemical and physical data
Formula	C10H14N5O8P
Molar mass	363.223 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES NC1=NC(=O)C2=C(N[C@@H]3O[C@@H]4COP(=O)(O)O[C@@H]4C(O)(O)[C@@H]3N2)N1;
	InChI InChI=1S/C10H14N5O8P/c11-9-14-6-3(7(16)15-9)12-4-8(13-6)22-2-1-21-24(19,20)23-5(2)10(4,17)18/h2,4-5,8,12,17-18H,1H2,(H,19,20)(H4,11,13,14,15,16)/t2-,4-,5+,8-/m1/s1; Key:CZAKJJUNKNPTTO-AJFJRRQVSA-N;

Fosdenopterin (or cyclic pyranopterin monophosphate, cPMP), sold under the brand name Nulibry, is a medication used to reduce the risk of death due to a rare genetic disease known as molybdenum cofactor deficiency type A.

The most common side effects include complications related to the intravenous line, fever, respiratory infections, vomiting, gastroenteritis, and diarrhea.

Fosdenopterin was approved for medical use in the United States in February 2021, It is the first medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of molybdenum cofactor deficiency type A. and in the European Union in September 2022. The US Food and Drug Administration considers it to be a first-in-class medication.