Primary Hyperoxaluria
4
Pipeline Programs
2
Companies
4
Clinical Trials
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
2100%
+ 2 programs with unclassified modality
On Market (1)
Approved therapies currently available
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Alnylam PharmaceuticalsCAMBRIDGE, MA
2 programs1
1
LumasiranPhase 3RNA Therapeutic1 trial
LumasiranN/ARNA Therapeutic1 trial
Active Trials
NCT04125472Approved For Marketing
Novo NordiskBAGSVAERD DENMARK, Denmark
2 programs1
1
nedosiranPhase 21 trial
DCR-PHXCPhase 11 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Alnylam PharmaceuticalsLumasiran
Novo Nordisknedosiran
Novo NordiskDCR-PHXC
Clinical Trials (4)
Total enrollment: 88 patients across 4 trials
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Start: Apr 2019Est. completion: Jul 202418 patients
Phase 3Completed
Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function
Start: Feb 2022Est. completion: Feb 202527 patients
Phase 2Completed
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
Start: Dec 2017Est. completion: Nov 201943 patients
Phase 1Completed
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
N/AApproved For Marketing
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
RNA Therapeutic is the dominant modality (100% of programs)
2 companies competing in this space