The award of priority review status to the company’s New Drug Application for lumasiran adds to the previous grants of Breakthrough Therapy and Pediatric Rare Disease Designations. The FDA has set a PDUFA action date of 12/3/2020, and also indicated that they are not currently planning an advisory committee meeting as part of the NDA review.
Additionally, the company’s Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the European Medicines Agency (EMA). Lumasiran was previously also granted an accelerated assessment by the EMA, which potentially reduces the agency’s evaluation time from 210 to 150 days.
Lumasiran has also previously received Orphan Drug Designations for the treatment of PH1 in the U.S. and Europe, and has received a Priority Medicines (PRIME) designation from the EMA.
Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1). HAO1 encodes glycolate oxidase (GO).Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. Lumasiran utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables quarterly subcutaneous maintainence dosing with increased potency and durability and a wide therapeutic index.