Fosdenopterin

Trade name: Nulibry

Small moleculeapprovedOrphan Drug FDABreakthrough Therapy FDA

Approved

Feb 26, 2021

CPMP may refer to:Committee for Proprietary Medicinal Products Certified Project Management Professional Core-Plus Mathematics Project Cyclic pyranopterin monophosphate or fosdenopterin Commissioning Process Management Professional (ASHRAE) — Wikipedia

Clinical trial activity

4 trials · 4 clinical orgs · 3 marketing orgs

Phase 1
2
Phase 2
2
Phase 3
1
Phase 4
0

Earliest trial started Aug 1, 2009 (NCT00957749)

Timeline

2000s

  1. Jan 1, 2009

    Earliest Phase 1 Sponsor(trial)

  2. Nov 5, 2009

    Orphan Drug Designation

2010s

  1. Jan 1, 2014

    Earliest Phase 2 Sponsor(trial)

2020s

  1. Feb 26, 2021

    Zydus Discovery DMCC — Earliest FDA Approval

  2. Feb 26, 2021

    Sentynl Theraps Inc — Marketing Organization

  3. Feb 26, 2021

    Bridge Biotherapeutics, Inc. — Marketing Organization

  4. Feb 26, 2021

    Zydus Discovery DMCC — NDA Secondary Org

  5. Feb 26, 2021

    Zydus Discovery DMCC — NDA Organization

Indications

Mechanism of action

  • Unspecified targetOTHER

    Unknown

    Substrate replacement therapy (exogenous source of cPMP)

Approval history

  • approvedPriority reviewBreakthroughFeb 26, 2021

Chemistry & pharmacology

Loading structure…

SMILES

Nc1nc(=O)c2c([nH]1)N[C@@H]1O[C@@H]3COP(=O)(O)O[C@@H]3C(O)(O)[C@@H]1N2
Mol. weight
363.22 g/mol
Lipinski Ro5
2 violation(s)
Rule of 3
No
Chirality
Single Stereoisomer
Inorganic
No
Polymer
No
Delivery
Parenteral
Availability
Prescription Only

Oral

No

Parenteral

Yes

Topical

No

First in class

Sources

Also known as

  • alxn1101
  • cpmp
  • cpmp
  • cyclic pyranopterin monophosphate

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT02629393Phase 2/Phase 3 (Phase 3)May 1, 2016Alexion
NCT02047461Phase 2Apr 1, 2014Alexion
NCT01894165Phase 1Jun 1, 2013Alexion, Parexel
NCT00957749Phase 1/Phase 2 (Phase 2)Aug 1, 2009Monash University, Orphatech Pharmaceuticals, GmbH
Showing 4 of 4 trials
Page 1 / 1

Organizations

Research & Development (4)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
AlexionFor profit3332013
Monash UniversityAcademic/Hospital1012009
Orphatech Pharmaceuticals, GmbHFor profit1112009
ParexelFor profit1012013
4 organizations
Page 1 / 1

Marketing (4)

OrganizationOrg typeRelationshipDate
Bridge Biotherapeutics, Inc.For profitMKTGFeb 26, 2021
Sentynl Theraps IncFor profitMKTGFeb 26, 2021
Zydus Discovery DMCCFor profitNDA2Feb 26, 2021
Zydus Discovery DMCCFor profitNDAFeb 26, 2021