Due to the lack of treatment options for the genetic
disease primary
hyperoxaluria (PH), including three subtypes PH1, PH2, and PH3, caused
by accumulation of oxalate forming kidney stones, there is an urgent
need for the development of a drug therapy aside from siRNA drug lumasiran
for patients with PH1. After the recent success of drug therapies
based on small interfering RNA (siRNA), nedosiran is currently being
developed for the treatment of three types of PH as a siRNA-based
modality. Through specific inhibition of lactate dehydrogenase enzyme,
the key enzyme in biosynthesis of oxalate in liver, phase 1, 2, and
3 clinical trials of nedosiran have achieved the desired primary end
point of reduction of urinary oxalate levels in patients with PH1.
More PH2 and PH3 patients need to be tested for efficacy. It has also
produced a favorable secondary end point on safety and toxicity in
PH patients. In addition to common injection site reactions that resolved
spontaneously, no severe nedosiran treatment-associated adverse events
were reported. Based on the positive results in the clinical studies,
nedosiran is a candidate siRNA drug to treat PH patients.
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