Drug updated on 10/21/2024
| Dosage Form | Injection (subcutaneous; 80 mg [0.5 mL], 128 mg [0.8 mL], 160 mg [1 mL]) |
| Drug Class | LDHA-directed small interfering RNAs |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for lowering urinary oxalate levels in children 9 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function, e.g., eGFR 30 mL/min/1.73 square meters.
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Summary
- This summary is based on the review of one randomized controlled trial(s). [1]
- Nedosiran significantly reduced 24-hour urinary oxalate (UOX) excretion, with an area under the curve (AUC) of +3507 (SE 788) in the nedosiran group compared to -1664 (SE 1190) in the placebo group, resulting in a difference of 5172 (95% CI 2929-7414; P < 0.001).
- In the PH1 subgroup, 64.7% achieved normal or near-normal Uox excretion on ≥2 consecutive visits starting at day 90, while 0% in the placebo group achieved this outcome (P < 0.001), indicating a favorable response to nedosiran.
- The PH2 subgroup did not demonstrate a consistent reduction in Uox, suggesting a less favorable or variable response to nedosiran.
- Nedosiran was generally safe and well tolerated, with injection-site reactions occurring in 9% of participants, all of which were mild and self-limiting.
- The safety profile of nedosiran indicates manageable adverse effects, with no significant safety concerns reported beyond the mild injection-site reactions.
- There is no population types or subgroups information available in the reviewed documents.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Rivfloza (nedosiran) Prescribing Information. | 2023 | Novo Nordisk Inc., Plainsboro, NJ |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| PHYOX2: a pivotal randomized study of nedosiran in primary hyperoxaluria type 1 or 2 | Data not availableSubjects F: null% M: null% | 2023 | Kidney International |
Sex Distribution:
Year:
2023
Source:Kidney International
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Clinical practice recommendations for primary hyperoxaluria: an expert consensus statement from ERKNet and OxalEurope. | 2023 | Nature Reviews. Nephrology |