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Mucopolysaccharidosis VI

0
Pipeline Programs
2
Companies
5
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
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

BioMarin Pharmaceutical
4 programs
N-acetylgalactosamine 4-sulfatasePHASE_11 trial
N-acetylgalactosamine 4-sulfatasePHASE_21 trial
N-acetylgalactosamine 4-sulfatasePHASE_31 trial
NaglazymePHASE_41 trial
Active Trials
NCT00048620CompletedEst. Nov 2005
NCT00048711CompletedEst. Jun 2006
NCT00104234Completed39Est. Oct 2006
+1 more trials
Inventiva
InventivaDAIX, France
1 program
OdiparcilPHASE_21 trial
Active Trials
NCT03370653Completed20Est. Oct 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
BioMarin PharmaceuticalNaglazyme
BioMarin PharmaceuticalN-acetylgalactosamine 4-sulfatase
InventivaOdiparcil
BioMarin PharmaceuticalN-acetylgalactosamine 4-sulfatase
BioMarin PharmaceuticalN-acetylgalactosamine 4-sulfatase

Clinical Trials (5)

Total enrollment: 63 patients across 5 trials

NCT00299000BioMarin PharmaceuticalNaglazyme

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

Start: May 2006Est. completion: Apr 20094 patients
Phase 4Completed
NCT00104234BioMarin PharmaceuticalN-acetylgalactosamine 4-sulfatase

Study of rhASB in Patients With Mucopolysaccharidosis VI

Start: Feb 2004Est. completion: Oct 200639 patients
Phase 3Completed
NCT03370653InventivaOdiparcil

A Study in MPS VI to Assess Safety and Efficacy of Odiparcil

Start: Dec 2017Est. completion: Oct 201920 patients
Phase 2Completed
NCT00048711BioMarin PharmaceuticalN-acetylgalactosamine 4-sulfatase

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Start: Mar 2002Est. completion: Jun 2006
Phase 2Completed
NCT00048620BioMarin PharmaceuticalN-acetylgalactosamine 4-sulfatase

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Start: Sep 2000Est. completion: Nov 2005
Phase 1Completed

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

2 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.