Sanfilippo Syndrome Type A (MPS IIIA)

2
Pipeline Programs
1
Companies
2
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
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Swedish Orphan Biovitrum
2 programs
2
SOBI003Phase 1/21 trial
SOBI003Phase 1/21 trial
Active Trials
NCT03811028CompletedEst. May 2021
NCT03423186CompletedEst. Oct 2019

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Swedish Orphan BiovitrumSOBI003
Swedish Orphan BiovitrumSOBI003

Clinical Trials (2)

NCT03811028Swedish Orphan BiovitrumSOBI003

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Start: Jan 2019Est. completion: May 2021
Phase 1/2Completed
NCT03423186Swedish Orphan BiovitrumSOBI003

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Start: Jun 2018Est. completion: Oct 2019
Phase 1/2Completed

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