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Wikipedia

Fosdenopterin

January 01, 1970

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]

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
  • 150829-29-1
PubChem CID
  • 135894389
DrugBank
  • DB16628
ChemSpider
  • 17221217
UNII
  • 4X7K2681Y7
KEGG
  • D11779
ChEMBL
  • ChEMBL2338675
CompTox Dashboard (EPA)
  • DTXSID90934067
Chemical and physical data
FormulaC10H14N5O8P
Molar mass363.223 g·mol−1
3D model (JSmol)
  • Interactive image
  • 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

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 edit

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

Mechanism of action edit

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 edit

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 edit

Legal status edit

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 edit

  1. ^ a b "Nulibry- fosdenopterin hydrobromide injection, powder, for solution". DailyMed. from the original on 20 June 2021. Retrieved 31 March 2021.
  2. ^ a b c d e f g h i j k "FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A". U.S. Food and Drug Administration (FDA) (Press release). 26 February 2021. from the original on 27 February 2021. Retrieved 26 February 2021.   This article incorporates text from this source, which is in the public domain.
  3. ^ a b c "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. from the original on 14 September 2021. Retrieved 13 September 2021.
  5. ^ a b Advancing Health Through Innovation: New Drug Therapy Approvals 2021 (PDF). U.S. Food and Drug Administration (FDA) (Report). 13 May 2022. from the original on 6 December 2022. Retrieved 22 January 2023.   This article incorporates text from this source, which is in the public domain.
  6. ^ DrugBank DB16628 . Accessed 5 March 2021.
  7. ^ Santamaria-Araujo JA, Fischer B, Otte T, Nimtz M, Mendel RR, Wray V, Schwarz G (April 2004). "The tetrahydropyranopterin structure of the sulfur-free and metal-free molybdenum cofactor precursor". The Journal of Biological Chemistry. 279 (16): 15994–9. doi:10.1074/jbc.M311815200. PMID 14761975.
  8. ^ Schwarz G, Santamaria-Araujo JA, Wolf S, Lee HJ, Adham IM, Gröne HJ, et al. (June 2004). "Rescue of lethal molybdenum cofactor deficiency by a biosynthetic precursor from Escherichia coli". Human Molecular Genetics. 13 (12): 1249–55. doi:10.1093/hmg/ddh136. PMID 15115759.
  9. ^ Tedmanson S (5 November 2009). "Doctors risk untried drug to stop baby's brain dissolving". TimesOnline. from the original on 22 June 2021. Retrieved 6 March 2021.
  10. ^ Schwahn BC, Van Spronsen FJ, Belaidi AA, Bowhay S, Christodoulou J, Derks TG, et al. (November 2015). "Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study". Lancet. 386 (10007): 1955–63. doi:10.1016/S0140-6736(15)00124-5. PMID 26343839. S2CID 21954888.
  11. ^ a b "Nulibry: Pending EC decision". European Medicines Agency. 22 July 2022. from the original on 28 July 2022. Retrieved 30 July 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  12. ^ "Nulibry Product information". Union Register of medicinal products. Retrieved 3 March 2023.

External links edit

  • "Fosdenopterin". Drug Information Portal. U.S. National Library of Medicine.

January 01, 1970
fosdenopterin, cyclic, pyranopterin, monophosphate, cpmp, sold, under, brand, name, nulibry, medication, used, reduce, risk, death, rare, genetic, disease, known, molybdenum, cofactor, deficiency, type, clinical, datatrade, namesnulibryother, namesprecursor, a. 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 FosdenopterinClinical dataTrade namesNulibryOther namesPrecursor Z ALXN1101License dataUS DailyMed FosdenopterinRoutes ofadministrationIntravenousATC codeA16AX19 WHO Legal statusLegal statusUS only 1 2 EU Rx only 3 IdentifiersIUPAC name 5 amino 11 11 14 trihydroxy 14 oxo 13 15 18 trioxa 2 4 6 9 tetraza 14l5 phosphatetracyclo 8 8 0 03 8 012 17 octadeca 3 8 4 dien 7 oneCAS Number150829 29 1PubChem CID135894389DrugBankDB16628ChemSpider17221217UNII4X7K2681Y7KEGGD11779ChEMBLChEMBL2338675CompTox Dashboard EPA DTXSID90934067Chemical and physical dataFormulaC 10H 14N 5O 8PMolar mass363 223 g mol 13D model JSmol Interactive imageSMILES NC1 NC O C2 C N C H 3O C H 4COP O O O C H 4C O O C H 3N2 N1InChI 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 s1Key CZAKJJUNKNPTTO AJFJRRQVSA N 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 Contents 1 Medical uses 2 Mechanism of action 3 History 4 Society and culture 4 1 Legal status 5 References 6 External linksMedical uses editFosdenopterin is indicated to reduce the risk of mortality in people with molybdenum cofactor deficiency MoCD type A 1 2 Mechanism of action editPeople 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 editFosdenopterin 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 editLegal status edit 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 edit a b Nulibry fosdenopterin hydrobromide injection powder for solution DailyMed Archived from the original on 20 June 2021 Retrieved 31 March 2021 a b c d e f g h i j k 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 nbsp This article incorporates text from this source which is in the public domain a b c 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 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 a b 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 nbsp This article incorporates text from this source which is in the public domain DrugBank DB16628 Accessed 5 March 2021 Santamaria Araujo JA Fischer B Otte T Nimtz M Mendel RR Wray V Schwarz G April 2004 The tetrahydropyranopterin structure of the sulfur free and metal free molybdenum cofactor precursor The Journal of Biological Chemistry 279 16 15994 9 doi 10 1074 jbc M311815200 PMID 14761975 Schwarz G Santamaria Araujo JA Wolf S Lee HJ Adham IM Grone HJ et al June 2004 Rescue of lethal molybdenum cofactor deficiency by a biosynthetic precursor from Escherichia coli Human Molecular Genetics 13 12 1249 55 doi 10 1093 hmg ddh136 PMID 15115759 Tedmanson S 5 November 2009 Doctors risk untried drug to stop baby s brain dissolving TimesOnline Archived from the original on 22 June 2021 Retrieved 6 March 2021 Schwahn BC Van Spronsen FJ Belaidi AA Bowhay S Christodoulou J Derks TG et al November 2015 Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A a prospective cohort study Lancet 386 10007 1955 63 doi 10 1016 S0140 6736 15 00124 5 PMID 26343839 S2CID 21954888 a b Nulibry Pending EC decision European Medicines Agency 22 July 2022 Archived from the original on 28 July 2022 Retrieved 30 July 2022 Text was copied from this source which is copyright European Medicines Agency Reproduction is authorized provided the source is acknowledged Nulibry Product information Union Register of medicinal products Retrieved 3 March 2023 External links edit Fosdenopterin Drug Information Portal U S National Library of Medicine Portal nbsp Medicine Retrieved from https en wikipedia org w index php title Fosdenopterin amp oldid 1189528470, wikipedia, wiki, book, books, library,

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