nedosiran (Rivfloza)

^[1]^[2]^[3]

Indications

FDA-approved to lower urinary oxalate levels in children >= 9 years of age & adults with primary hyperoxaluria type 1 with relatively preserved renal function

once monthly SQ injection into the abdomen or upper thigh
- 160 mg adults & children or adolecents >= 50 kg
- 3.3 mg/kg children < 50 kg, not toexceed 128 mg (Vial, dose volume rounded to nearest 0.1 mL)

* single-dose vial or Pre-filled Syringe

small inhibitory RNA (siRNAP reduces levels of hepatic lactate dehydrogenase (LDH) the enzyme responsible for the conversion of glyoxylate to oxalate

↑ Goldfarb DS, Lieske JC, Groothoff J et al Nedosiran in primary hyperoxaluria subtype 3: results from a phase I, single-dose study (PHYOX4). Urolithiasis. 2023 Apr 28;51(1):80. PMID: https://www.ncbi.nlm.nih.gov/pubmed/37118061 PMCID: PMC10147791 Free PMC article. Clinical Trial.
↑ Hoppe B, Koch A, Cochat P et al Safety, pharmacodynamics, and exposure-response modeling results from a first-in-human phase 1 study of nedosiran (PHYOX1) in primary hyperoxaluria. Kidney Int. 2022 Mar;101(3):626-634. Epub 2021 Sep 2. PMID: https://www.ncbi.nlm.nih.gov/pubmed/34481803 Free article. Clinical Trial.
↑ HIGHLIGHTS OF PRESCRIBING INFORMATION RIVFLOZA<TM> (nedosiran) injection, for subcutaneous use https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf