Recent Updates
Recently added Catalysts

nedosiran

Phase 2

Primary Hyperoxaluria | Small molecule | Other |Novo Nordisk A/S|Last Updated: Apr 16, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment27
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05001269Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal FunctionPHASE2 COMPLETED 27 — — Feb 22, 2022Feb 5, 2025Apr 16, 202613 United States, Canada +9
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Percent Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups
Baseline (Week 0), Month 6

This outcome measure reported percent change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed primary hyperoxaluria type 1 (PH1), primary hyperoxaluria type 2 (PH2), or primary hyperoxaluria type 3 (PH3) subgroups.

Absolute Change From Baseline to Month 6 in Spot Urinary Oxalate-to-creatinine Ratio in PH1, PH2, or PH3 Participant Subgroups
Baseline (Week 0), Month 6

This outcome measure reported absolute change from baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in pediatric participants (birth to 11 years of age) with genetically confirmed PH1, PH2, or PH3 subgroups.

Secondary Endpoints
Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From baseline (Week 0) up to Month 6
Number of Treatment Emergent Adverse Events and Serious Adverse Events-Nature
From baseline (Week 0) up to Month 6
Change From Baseline in 12-lead Electrocardiogram (ECG)- ECG Mean Heart Rate
Baseline (Week 0), Month 6
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Open-Label DCR-PHXCEXPERIMENTALOpen-Label monthly subcutaneous injection of DCR-PHXC based on age and weight.
Interventions
NameTypeDescription
nedosiranDRUGMonthly subcutaneous dosing throughout study period
Unlock Study Design Details
Eligibility Criteria
Age RangeN/A — 11 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: 1. Birth to 11 years of age inclusive, at the time of signing the informed consent. 2. Documented diagnosis of PH1 or PH2 or PH3 confirmed by genotyping (historically available genotype information is acceptable for study eligibility). 3. Average spot Uox to creatinine ratio at ...

Countries:United StatesCanadaGermanyItalyJapanLebanonPolandSpainTurkey (Türkiye)United Arab EmiratesUnited Kingdom
Unlock Eligibility Criteria