Primary Hyperoxaluria Type 1 (PH1)
3
Pipeline Programs
2
Companies
4
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
1
0
1
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
1100%
+ 3 programs with unclassified modality
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Novo NordiskBAGSVAERD DENMARK, Denmark
3 programs1
1
DCR-PHXCPhase 31 trial
DCR-PHXCPhase 21 trial
NedosiranN/A1 trial
Active Trials
NCT05993416No Longer Available
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Novo NordiskDCR-PHXC
Novo NordiskDCR-PHXC
Alnylam PharmaceuticalsLumasiran
Clinical Trials (4)
Total enrollment: 162 patients across 4 trials
Long Term Extension Study in Patients With Primary Hyperoxaluria
Start: Jul 2019Est. completion: Apr 203075 patients
Phase 3Active Not Recruiting
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
Start: Oct 2019Est. completion: Jun 202135 patients
Phase 2Completed
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
Start: Mar 2016Est. completion: Jan 201952 patients
Phase 1/2Completed
Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
N/ANo Longer Available
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