In this connection, the RNAi therapeutics company completed the rolling submission of a new drug application (NDA) for lumasiran with the regulator.
Considered to be an ultra-rare, life-threatening disease, primary hyperoxaluria type 1 affects the kidneys and other vital organs in infants, children, and adults.
Lumasiran, which is an investigational RNAi therapeutic designed to target glycolate oxidase (GO), was previously given pediatric rare disease designation, orphan drug designation, and breakthrough therapy designation by the FDA for the treatment of primary hyperoxaluria type 1.
The designations from the FDA were granted on data that showed a significant reduction in urinary oxalate, which is considered to be the main toxic metabolite responsible for the clinical manifestations of the disease.
Alnylam Pharmaceuticals has also submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for the investigational RNAi therapeutic in the same indication.
In the European Union, lumasiran holds the priority medicines (PRIME) designation and also the orphan drug designation.
The RNAi therapeutic candidate has also been given an accelerated assessment by the EMA.
Alnylam Pharmaceuticals vice president Pritesh Gandhi said: “PH1 causes a progressive decline in kidney function and can lead to end-stage renal disease, at which point patients need intensive dialysis until they are able, and eligible, to receive dual liver/kidney transplantation.
“Given the unmet need in PH1 and the encouraging lumasiran Phase 3 data, Alnylam maintained its commitment to meet our target dates for timely NDA and MAA submissions, even under the challenging prevailing circumstances. We now look forward to working closely with the FDA and EMA to bring this innovative medicine to patients and their families.”
In the ILLUMINATE-A phase 3 trial, the RNAi therapeutic candidate met the primary efficacy endpoint and also all the tested secondary endpoints. The primary efficacy endpoint of the late-stage trial was the percent change from baseline, in 24-hour urinary oxalate excretion averaged across months three to six, in comparison to placebo.