Fosdenopterin

Summary

Fosdenopterin (CHEMBL2338675) is an approved small molecule (ATC A16AX19); indicated across 1 condition including inborn metal metabolism disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A16AX19
• Indications: 1 condition
• Clinical trials: 1
• Chemistry: 363.22 Da · C10H14N5O8P

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2338675
Name	Fosdenopterin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	135463437
ChEBI	CHEBI:60210
ATC	A16AX19
Molecular formula	C10H14N5O8P
Molecular weight	363.22
InChIKey	CZAKJJUNKNPTTO-AJFJRRQVSA-N

SMILES: C1[C@@H]2[C@@H](C([C@H]3[C@@H](O2)NC4=C(N3)C(=O)NC(=N4)N)(O)O)OP(=O)(O1)O

IUPAC name: (1R,10R,12S,17R)-5-amino-11,11,14-trihydroxy-14-oxo-13,15,18-trioxa-2,4,6,9-tetraza-14lambda5-phosphatetracyclo[8.8.0.03,8.012,17]octadeca-3(8),4-dien-7-one

ChEBI definition: A linear-fused organic heterotetracyclic compound consisting of a [1,3,2]dioxaphosphinane fused to a pyran ring which is in turn fused to a pteridine ring system. Molybdenum cofactor biosynthesis intermediate. Dehydrated derivative known as precursor Z.

Other ChEBI roles (chemical / environmental): Escherichia coli metabolite.

Also known as: ALXN-1101 ANHYDROUS FREE BASE, ALXN1101, C(pmp), Cyclic pyranopterin monophosphate, Fosdenopterin, Fosdenopterina, Fosdenopterine, Precursor z, FOSDENOPTERIN

Parent form; salt/anhydrous children: CHEMBL2338674, CHEMBL4594300

Patent coverage: 14 distinct patent families (40 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 28 (70%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
inborn metal metabolism disorder	1	MONDO:0004689	MONDO:0004689

Clinical trials

Total trials: 1.

Phase distribution

Phase	Trials
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01894165	PHASE1	COMPLETED	Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).