Mucopolysaccharidosis VI
5
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
1
0
2
0
1
1
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
BioMarin PharmaceuticalNOVATO, CA
4 programs1
1
1
1
NaglazymePhase 41 trial
N-acetylgalactosamine 4-sulfatasePhase 31 trial
N-acetylgalactosamine 4-sulfatasePhase 21 trial
N-acetylgalactosamine 4-sulfatasePhase 11 trial
Active Trials
+1 more trials
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: 43 patients across 5 trials
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
Study of rhASB in Patients With Mucopolysaccharidosis VI
Start: Feb 2004Est. completion: Oct 200639 patients
Phase 3Completed
A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
Start: Dec 2017Est. completion: Oct 2019
Phase 2Completed
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
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
1 late-stage (Phase 3) programs — potential near-term approvals
2 companies competing in this space