Alnylam Pharmaceuticals and the FDA agree on pivotal study design for lumasiran for the treatment of primary hyperoxaluria type 1.

Alnylam Pharmaceuticals, Inc. , the leading RNAi therapeutics company, announced that the Company has reached alignment with the FDA on a pivotal study design for lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1 (PH1). The Company and the FDA have aligned on a primary endpoint for the pivotal study based on reduction of urinary oxalate at six months, a biomarker directly linked to the pathophysiology of PH1 and known to be well correlated with disease progression. In addition, Alnylam and the FDA have aligned on a study size of approximately 25 patients with PH1.

Based on the discussions with the FDA, the Company is on track to start the Phase III study in mid-2018 and is now guiding that it expects to report topline results in 2019 and, if positive, to submit an NDA in early 2020.