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Primary Hyperoxaluria Type 1

3
Pipeline Programs
3
Companies
11
Clinical Trials
2 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
2
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
1100%
+ 4 programs with unclassified modality

On Market (1)

Approved therapies currently available

Alnylam Pharmaceuticals
OXLUMOApproved
lumasiran
Alnylam Pharmaceuticals
subcutaneous2020

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Alnylam Pharmaceuticals
2 programs
1
1
OXLUMO(lumasiran)Phase 3RNA Therapeutic5 trials
ALN-GO1 (Primary Hyperoxaluria registry)N/A
Active Trials
NCT04125472Approved For Marketing
NCT06225544Recruiting50Est. Mar 2025
NCT05161936Terminated2Est. Nov 2022
+2 more trials
Biocodex
BiocodexFrance - Gentilly
1 program
1
Stiripentol Oral CapsulePhase 31 trial
Active Trials
NCT06465472Not Yet Recruiting42Est. Aug 2030
Novo Nordisk
Novo NordiskBAGSVAERD DENMARK, Denmark
2 programs
DCR-PH1PHASE_11 trial
DCR-PHXCPHASE_21 trial
Active Trials
NCT02795325Terminated41Est. Oct 2016
NCT04580420Recruiting28Est. Jan 2032

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
BiocodexStiripentol Oral Capsule
Alnylam Pharmaceuticalslumasiran
Alnylam Pharmaceuticalslumasiran
Alnylam Pharmaceuticalslumasiran
Alnylam Pharmaceuticalslumasiran
Alnylam Pharmaceuticalslumasiran
Novo NordiskDCR-PHXC
Alnylam Pharmaceuticalslumasiran
Alnylam Pharmaceuticalslumasiran
Novo NordiskDCR-PH1

Clinical Trials (11)

Total enrollment: 313 patients across 11 trials

NCT06465472BiocodexStiripentol Oral Capsule

Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3

Start: Aug 2024Est. completion: Aug 203042 patients
Phase 3Not Yet Recruiting

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

Start: Jan 2020Est. completion: Jun 202521 patients
Phase 3Completed

A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

Start: Apr 2019Est. completion: Jul 202418 patients
Phase 3Completed

A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

Start: Nov 2018Est. completion: Jan 202439 patients
Phase 3Completed

Lumasiran in Hyperoxalaemic Patients on Haemodialysis

Start: Apr 2024Est. completion: Mar 202550 patients
Phase 2Recruiting

A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels

Start: Jan 2022Est. completion: Nov 20222 patients
Phase 2Terminated

Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD

Start: Apr 2021Est. completion: Jan 203228 patients
Phase 2Recruiting

An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1

Start: Apr 2018Est. completion: Feb 202320 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

A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)

Start: May 2016Est. completion: Oct 201641 patients
Phase 1Terminated

Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1

N/AApproved For Marketing

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 313 patients
3 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.