Alnylam Announces that the United Kingdom’s MHRA Grants Early Access to Lumasiran - Seite 2

The MHRA’s decision is based on the evaluation of the effects of lumasiran in PH1 patients and its safety profile, including data from the ILLUMINATE-A Phase 3 study. The results of the ILLUMINATE-A study were presented at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress, held June 6-9, 2020 as a virtual event.

A marketing authorization application (MAA) for lumasiran has been submitted to the European Medicines Agency (EMA) in April 2020 and was granted Accelerated Assessment. Lumasiran previously received Priority Medicines (PRIME) designation. The EC decision, which will apply to the UK, is expected in late 2020. In addition, Alnylam filed a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA). The FDA has granted a Priority Review for the NDA and has set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA).

About Lumasiran

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 subcutaneous dosing with increased potency and durability and a wide therapeutic index. Lumasiran has received both EU and U.S. Orphan Drug Designations, Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) and Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). The safety and efficacy of lumasiran are under evaluation by the FDA and EMA.

About ILLUMINATE-A Phase 3 Study

ILLUMINATE-A (NCT03681184) is a six-month randomized, double-blind, placebo-controlled, global, multicenter Phase 3 study (with a 54-month extension period) to evaluate the efficacy and safety of lumasiran in 39 patients with a documented diagnosis of PH1. Patients were randomized 2:1 to receive three monthly doses of lumasiran or placebo followed by quarterly maintenance doses at 3 mg/kg. The primary endpoint was the percent change in 24-hour urinary oxalate excretion from baseline to the average of months 3 to 6 in the patients treated with lumasiran as compared to placebo. Treatment arms were stratified at randomization based upon mean 24-hour urinary oxalate during screening (≤ 1.7 or > 1.7 mmol/24hr/1.73m²). Key secondary and exploratory endpoints were designed to evaluate additional measures of urinary oxalate, plasma oxalate, estimated glomerular filtration rate (eGFR), nephrocalcinosis, renal stone events, safety and tolerability.