Promising results for nedosiran in primary hyperoxaluria presented during Kidney Week 2023

Nedosiran, an RNA interference therapy, has shown promising results in treating primary hyperoxaluria (PH), a rare genetic disorder, according to data presented at Kidney Week 2023 Annual Meeting.

The 30-month interim analysis of the PHYOX3 study indicates that nedosiran was well-tolerated in patients with PH1 and resulted in a sustained reduction in urinary oxalate excretion, a key factor in preventing kidney damage.

The study included 13 participants with PH1. The estimated glomerular filtration rate (eGFR) was 77.6 mL/min/1.73m² at baseline and remained stable, ranging from 62 to 84.2 mL/min/1.73m² over the 30-month period.

One of the most significant findings was the sustained reduction in mean urinary oxalate (Uox) excretion, which showed a reduction of over 60% from baseline to month 30. At each visit, a substantial majority (76.9%) of participants achieved either normal or near-normal 24-hour Uox excretion levels.

In terms of safety, all participants experienced at least 1 adverse event (AE), the majority of which were of mild or moderate severity and deemed treatment-related in 76.9% of cases. Only 2.5% of the 398 total injections administered in the study resulted in injection-site reactions. Notably, there were no deaths or study discontinuations attributed to adverse events.

Three serious AEs were reported, although they were determined to be unrelated to the treatment. Ongoing analysis will further explore the long-term effects of nedosiran on kidney function.

Reference
Jaap G, et al. Long-Term Nedosiran Safety and Efficacy in Primary Hyperoxaluria Type 1 (PH1): Interim Analysis of PHYOX3. Presented at: Kidney Week 2023.