Pritesh J. Gandhi, PharmD, of Alnylam Pharmaceuticals discusses the company’s hope that lumasiran obtains FDA approval to treat primary hyperoxalaluria type 1 (PH1). A PDUFA date is set for December 3, 2020.

PH1 is a rare genetic disease in which excessive oxalate production leads to painful and recurrent kidney stones. These recurrent stones increase the chance of hematuria, urinary tract infections, and end stage renal disease (ESRD). 

Lumasiran is an RNAi drug that targets hydroxyacid oxidase 1 (HAO1), the gene that encodes glycolate oxidase. By silencing HAO1 and reducing glycolate oxidase, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. 

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