Homocystinuria
3
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
1
0
0
2
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Travere TherapeuticsSAN DIEGO, CA
3 programs1
2
PegtibatinasePhase 31 trial
PegtibatinasePhase 31 trial
PegtibatinasePhase 1/21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Travere TherapeuticsPegtibatinase
Travere TherapeuticsPegtibatinase
Travere TherapeuticsPegtibatinase
Clinical Trials (3)
Total enrollment: 202 patients across 3 trials
A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
Start: Apr 2024Est. completion: Jan 2027100 patients
Phase 3Enrolling By Invitation
A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment
Start: Dec 2023Est. completion: Jan 202670 patients
Phase 3Active Not Recruiting
Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)
Start: Jan 2019Est. completion: Dec 202632 patients
Phase 1/2Active Not Recruiting
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 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space