Mucopolysaccharidosis IV A
0
Pipeline Programs
1
Companies
3
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
1 companies ranked by most advanced pipeline stage
BioMarin PharmaceuticalNOVATO, CA
3 programsElosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]N/A1 trial
Enzyme testingN/A1 trial
BMN 110 - WeeklyPHASE_31 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
BioMarin PharmaceuticalBMN 110 - Weekly
BioMarin PharmaceuticalElosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
BioMarin PharmaceuticalEnzyme testing
Clinical Trials (3)
Total enrollment: 202 patients across 3 trials
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Start: Jul 2011Est. completion: Jun 2016173 patients
Phase 3Completed
Natural History of Atypical Morquio A Disease
Start: Feb 2018Est. completion: Sep 20257 patients
N/ACompleted
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Start: Oct 2012Est. completion: Jun 201522 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 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.