Target PH1 at the source of oxalate overproduction with OXLUMO1
OXLUMO is the first and only FDA-approved treatment for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in children and adults.1-3
As an siRNA targeted specifically at the liver, OXLUMO harnesses the natural RNAi pathway to reduce hepatic oxalate production.1
RNA, ribonucleic acid; RNAi, RNA interference; siRNA, small interfering RNA.
A metabolic defect causes continuous oxalate overproduction in PH14,5
In primary hyperoxaluria type 1 (PH1), mutations in the AGXT gene impair function of a liver enzyme called AGT4,5:
- AGT function is impaired4,5
- Oxalate is continuously overproduced as a result5,6
- Continuous oxalate overproduction causes the symptoms of PH14
AGXT, alanine:glyoxylate aminotransferase; AGT, alanine:glyoxylate aminotransferase.
OXLUMO reduces oxalate production1
OXLUMO decreases levels of glycolate oxidase (GO), an enzyme upstream of AGT. This reduces production of oxalate by the liver.1
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References: 1. OXLUMO (lumasiran) [Package Insert]. Cambridge, MA. Alnylam Pharmaceuticals, Inc. 2. Hoppe B. Nat Rev Nephrol. 2012;8(8):467-475. 3. Liebow A, Li X, Racie T, et al. J Am Soc Nephrol. 2017;28(2):494-503. 4. Milliner DS, Harris PC, Cogal AG, et al. https://www.ncbi.nlm.nih.gov/books/NBK1283/. Updated November 30, 2017. Accessed November 16, 2020. 5. Cochat P, Hulton SA, Acquaviva C, et al. Nephrol Dial Transplant. 2012;27(5):1729-1736. 6. Cochat P, Rumsby G. N Engl J Med. 2013;369(7):649-658.