Alnylam Pharmaceuticals, an important company focused on RNAi therapies, announced that the Company has reached alignment with the US Food and Drug Administration (FDA) on a pivotal study project for lumasiran, an experimental RNAi for the treatment of primary hyperoxaluria type 1 (PH1). The Company and the FDA aligned on a primary endpoint for the registration study based on the reduction of six-month urinary oxalate, a biomarker directly related to the pathophysiology of PH1 and known to be well correlated with the progression of the disease. Furthermore, Alnylam and FDA have aligned themselves on a study size of about 25 patients with PH1. Based on discussions with the FDA, the Company is on track to begin the Phase 3 study in mid-2018, with plans to report topline results in 2019 and, if positive, to present an NDA at the beginning in 2020. Lumasiran was recently granted the designation of Breakthrough Therapy and Priority Medicines (PRIME) by the European Medicines Agency (EMA). Lumasiran offers hope to patients with PH1 who face health challenges. Alnylam’s alignment with the FDA key study project is an important step forward for patients suffering from this serious disease, as it recognizes the urgency of unmet need for effective treatment for PH1 and the potential for lumasiran to meet this necessity. The ongoing Phase I / IIb study is designed as a randomized, single-blind, placebo-controlled study. Patients with PH1, aged six or more, were enrolled.

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