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With trial win, Alnylam to seek approval for first treatment of rare kidney disease

Credit: Alnylam Pharmaceuticals

Dive Brief:

  • Alnylam Pharmaceuticals on Tuesday said its experimental drug lumasiran succeeded in a Phase 3 study of patients with ultra-rare kidney disease, leading the biotech to plan regulatory submissions in the U.S. and Europe in early 2020 for the RNA interference-based medicine.

  •  The late-stage trial included about 30 patients with primary hyperoxaluria type 1, a genetic disease that causes recurrent kidney stones and damages organs over time, often requiring dialysis and transplants. Alnylam will seek approval based on lumasiran's benefit on urinary excretion levels of a metabolite called oxalate, a biomarker cleared last year by the Food and Drug Administration.

  • Alnylam has led the RNA interference field, but one of its closest rivals may soon threaten lumasiran with another RNAi treatment for primary hyperoxaluria. Dicerna Pharmaceuticals has an ongoing pivotal study for DCR-PHXC, testing patients with both Type 1 and Type 2 of the disease.

Written by: Andrew Dunn

Published on: Dec. 17, 2019

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