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Fosdenopterin

Medication From Wikipedia, the free encyclopedia

Fosdenopterin

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]

Quick Facts Clinical data, Trade names ...
Fosdenopterin
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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
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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]

Medical uses

Fosdenopterin is indicated to reduce the risk of mortality in people with molybdenum cofactor deficiency (MoCD) type A.[1][2]

Mechanism of action

People with molybdenum cofactor deficiency type A cannot produce cyclic pyranopterin monophosphate (cPMP) in their body.[2] Fosdenopterin is an intravenous medication that replaces the missing cPMP.[2][6] cPMP is a precursor to molybdopterin, which is required for the enzyme activity of sulfite oxidase, xanthine dehydrogenase/oxidase and aldehyde oxidase.[7]

History

Fosdenopterin was developed at the German universities TU Braunschweig and the University of Cologne.[8][9]

The effectiveness of fosdenopterin for the treatment of MoCD-A was demonstrated in thirteen treated participants compared to eighteen matched, untreated participants.[2][10] The participants treated with fosdenopterin had a survival rate of 84% at three years, compared to 55% for the untreated participants.[2]

The U.S. Food and Drug Administration (FDA) granted the application for fosdenopterin priority review, breakthrough therapy, and orphan drug designations along with a rare pediatric disease priority review voucher.[2][5] The FDA granted the approval of Nulibry to Origin Biosciences, Inc., in February 2021.[2]

Society and culture

On 21 July 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Nulibry, intended for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.[11] The applicant for this medicinal product is Comharsa Life Sciences Ltd.[11] Fosdenopterin was approved for medical use in the European Union in September 2022.[3][12]

References

External links

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