Fosdenopterin Explained

Tradename:Nulibry
Dailymedid:Fosdenopterin
Routes Of Administration:Intravenous
Atc Prefix:A16
Atc Suffix:AX19
Legal Us:Rx-only
Legal Us Comment:[1]
Legal Eu:Rx-only
Legal Eu Comment:[2]
Cas Number:150829-29-1
Pubchem:135894389
Drugbank:DB16628
Chemspiderid:17221217
Unii:4X7K2681Y7
Kegg:D11779
Chembl:2338675
Synonyms:Precursor Z, ALXN1101
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
C:10
H:14
N:5
O:8
P:1
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
Stdinchi: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
Stdinchikey: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.[3]

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,[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. and in the European Union in September 2022. 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.

Mechanism of action

People with molybdenum cofactor deficiency type A cannot produce cyclic pyranopterin monophosphate (cPMP) in their body. Fosdenopterin is an intravenous medication that replaces the missing cPMP.[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.[10] The participants treated with fosdenopterin had a survival rate of 84% at three years, compared to 55% for the untreated participants.

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. The FDA granted the approval of Nulibry to Origin Biosciences, Inc., in February 2021.

Society and culture

Legal status

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. 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.[12]

External links

Notes and References

  1. Web site: Nulibry- fosdenopterin hydrobromide injection, powder, for solution . DailyMed . 31 March 2021 . 20 June 2021 . https://web.archive.org/web/20210620214011/https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=4f67cc4e-84ed-4f4e-a5d9-6ffbfb84eddd . live .
  2. Web site: Nulibry EPAR . European Medicines Agency (EMA) . 18 July 2022 . 14 October 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  3. FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A . U.S. Food and Drug Administration (FDA) . 26 February 2021 . 26 February 2021 . 27 February 2021 . https://web.archive.org/web/20210227102226/http://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-molybdenum-cofactor-deficiency-type . live .
  4. Web site: Drug Approval Package: Nulibry . U.S. Food and Drug Administration (FDA) . 26 March 2021 . 13 September 2021 . 14 September 2021 . https://web.archive.org/web/20210914043022/https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214018Orig1s000TOC.cfm . live .
  5. Advancing Health Through Innovation: New Drug Therapy Approvals 2021 . U.S. Food and Drug Administration (FDA) . 13 May 2022 . PDF . 22 January 2023 . 6 December 2022 . https://web.archive.org/web/20221206210020/https://www.fda.gov/media/155227/download . live .
  6. . Accessed 5 March 2021.
  7. Santamaria-Araujo JA, Fischer B, Otte T, Nimtz M, Mendel RR, Wray V, Schwarz G . The tetrahydropyranopterin structure of the sulfur-free and metal-free molybdenum cofactor precursor . The Journal of Biological Chemistry . 279 . 16 . 15994–9 . April 2004 . 14761975 . 10.1074/jbc.M311815200 . free .
  8. Schwarz G, Santamaria-Araujo JA, Wolf S, Lee HJ, Adham IM, Gröne HJ, Schwegler H, Sass JO, Otte T, Hänzelmann P, Mendel RR, Engel W, Reiss J . 6 . Rescue of lethal molybdenum cofactor deficiency by a biosynthetic precursor from Escherichia coli . Human Molecular Genetics . 13 . 12 . 1249–55 . June 2004 . 15115759 . 10.1093/hmg/ddh136 . free .
  9. News: Tedmanson S . Doctors risk untried drug to stop baby's brain dissolving . TimesOnline . 5 November 2009 . 6 March 2021 . 22 June 2021 . https://web.archive.org/web/20210622174050/https://www.thetimes.co.uk/article/doctors-risk-untried-drug-to-stop-babys-brain-dissolving-rbp08z32pwr . live .
  10. Schwahn BC, Van Spronsen FJ, Belaidi AA, Bowhay S, Christodoulou J, Derks TG, Hennermann JB, Jameson E, König K, McGregor TL, Font-Montgomery E, Santamaria-Araujo JA, Santra S, Vaidya M, Vierzig A, Wassmer E, Weis I, Wong FY, Veldman A, Schwarz G . 6 . Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study . Lancet . 386 . 10007 . 1955–63 . November 2015 . 26343839 . 10.1016/S0140-6736(15)00124-5 . 21954888 .
  11. Web site: Nulibry: Pending EC decision . European Medicines Agency . 22 July 2022 . 30 July 2022 . 28 July 2022 . https://web.archive.org/web/20220728174918/https://www.ema.europa.eu/en/medicines/human/summaries-opinion/nulibry . live . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  12. Web site: Nulibry Product information . Union Register of medicinal products . 3 March 2023.

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