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Pantothenate Kinase-Associated Neurodegeneration

1
Pipeline Programs
4
Companies
4
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Chiesi
ChiesiBrazil - Santana de Parnaíba
2 programs
1
Deferiprone oral solutionPhase 31 trial
DeferiproneN/A1 trial
Active Trials
NCT02635841No Longer Available
NCT02174848Completed68Est. Mar 2018
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
CoA-ZN/A1 trial
Active Trials
NCT04182763Completed77Est. Jan 2025
Oregon Therapeutics
Oregon TherapeuticsFrance - Paris
1 program
CoA-ZN/A
Travere Therapeutics
1 program
FosmetpantotenatePHASE_31 trial
Active Trials
NCT03041116Terminated84Est. Dec 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Travere TherapeuticsFosmetpantotenate
ChiesiDeferiprone oral solution
Human BioSciencesCoA-Z

Clinical Trials (4)

Total enrollment: 229 patients across 4 trials

NCT03041116Travere TherapeuticsFosmetpantotenate

Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants

Start: Jul 2017Est. completion: Dec 201984 patients
Phase 3Terminated
NCT02174848ChiesiDeferiprone oral solution

Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration

Start: Jun 2014Est. completion: Mar 201868 patients
Phase 3Completed
NCT02635841ChiesiDeferiprone

Compassionate Use of Deferiprone in Patients With PKAN

N/ANo Longer Available
NCT04182763Human BioSciencesCoA-Z

CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)

Start: Dec 2019Est. completion: Jan 202577 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
4 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.