Alnylam Reports Alignment With FDA On Accelerated Development Path For Lumasiran

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY) announced it has reached alignment with the U.S. FDA on a pivotal study design for lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1, or 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. Alnylam and the FDA have also aligned on a study size of approximately 25 patients with PH1.

Based on the discussions with the FDA, the company said it is on track to start the Phase 3 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.

Lumasiran was recently granted Breakthrough Therapy Designation from the FDA and Priority Medicines (PRIME) designation from the European Medicines Agency.