Primary Hyperoxaluria Type 1
4
Pipeline Programs
2
Companies
3
Clinical Trials
1 recruiting
1
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
1
0
1
1
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
1100%
+ 2 programs with unclassified modality
On Market (1)
Approved therapies currently available
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Novo NordiskBAGSVAERD DENMARK, Denmark
2 programs1
1
DCR-PHXCPhase 21 trial
DCR-PH1Phase 11 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Alnylam PharmaceuticalsLumasiran
Novo NordiskDCR-PHXC
Novo NordiskDCR-PH1
Clinical Trials (3)
Total enrollment: 90 patients across 3 trials
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
Start: Jan 2020Est. completion: Jun 202521 patients
Phase 3Completed
Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
Start: Apr 2021Est. completion: Jan 203228 patients
Phase 2Recruiting
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
Start: May 2016Est. completion: Oct 201641 patients
Phase 1Terminated
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
1 actively recruiting trials targeting 90 patients
2 companies competing in this space