Fosdenopterin

Fosdenopterin
Clinical data
Trade namesNulibry
Other namesPrecursor Z, ALXN1101
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
  • 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
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC10H14N5O8P
Molar mass363.223 g·mol−1
3D model (JSmol)
  • 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=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.[2]

The most common side effects include complications related to the intravenous line, fever, respiratory infections, vomiting, gastroenteritis, and diarrhea.[2]

Fosdenopterin was approved for medical use in the United States in February 2021,[4] It is the first medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of molybdenum cofactor deficiency type A.[2] and in the European Union in September 2022.[3] The US Food and Drug Administration considers it to be a first-in-class medication.[5]

  1. ^ "Nulibry- fosdenopterin hydrobromide injection, powder, for solution". DailyMed. Archived from the original on 20 June 2021. Retrieved 31 March 2021.
  2. ^ a b c d "FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A". U.S. Food and Drug Administration (FDA) (Press release). 26 February 2021. Archived from the original on 27 February 2021. Retrieved 26 February 2021. Public Domain This article incorporates text from this source, which is in the public domain.
  3. ^ a b "Nulibry EPAR". European Medicines Agency (EMA). 18 July 2022. Retrieved 14 October 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  4. ^ "Drug Approval Package: Nulibry". U.S. Food and Drug Administration (FDA). 26 March 2021. Archived from the original on 14 September 2021. Retrieved 13 September 2021.
  5. ^ Advancing Health Through Innovation: New Drug Therapy Approvals 2021 (PDF). U.S. Food and Drug Administration (FDA) (Report). 13 May 2022. Archived from the original on 6 December 2022. Retrieved 22 January 2023. Public Domain This article incorporates text from this source, which is in the public domain.