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Alnylam's lumasiran hits main Phase 3 goal in rare renal disease primary hyperoxaluria type 1

Published on 09/06/20 at 12:55pm
Image credit: Business Wire

Alnylam has presented promising new Phase 3 data at the European Renal Association-European Dialysis and Transplant Association International Congress for its RNA interference (RNAi) drug lumasiran in the treatment of primary hyperoxaluria type 1 (PH1), an ultra-rare kidney and urinary tract disease.

The condition is often characterised by painful and recurrent kidney stones and nephrocalcinosis, and can be fatal due to accumulation of crystalised oxalate in the eyes, skin, bones and heart.

It can only currently be cured through a dual liver and kidney transplant; donors for such a potentially dangerous procedure are in short supply, and patients who do undergo the procedure then require immunosuppression for the rest of their lives. Lumasiran works to treat the condition by targeting hydroxyacid oxidase 1 (HAO1) responsible for encoding glycolate oxidase, in turn stopping the production of oxalate.

The data presented by Alynylam showed that use of lumasiran led to a mean reduction of 53.5% in urinary oxalate levels compared to placebo, meeting the trial’s primary endpoint. This mean reduction was recorded as 64.5% when taken relative to baseline.

It was reported that the study also met all of its secondary goals, including 84% and 52% of participants achieving near-normalisation or normalisation, respectively, of urinary oxalate levels. No patients in the placebo group achieved the same.

“The substantial and sustained reductions in urinary and plasma oxalate reported demonstrate that lumasiran addressed the underlying pathophysiology of PH1 by reducing the production of the toxic metabolite responsible for the clinical manifestations of this serious and progressive disease,” explained Akshay Vaishnaw, President of R&D at Alnylam. “Thus, we believe lumasiran has the potential to have a favorable impact on disease manifestations, including nephrocalcinosis and renal stones, and overall disease progression, which we are continuing to evaluate in the ongoing ILLUMINATE programme.”

Lumasiran has chalked up a number of designations from global regulators, including Orphan Drug Designation from the FDA and EMA, as well as Breakthrough Therapy Designation and Priority Medicines designation from these two agencies. Alnylam noted it expects a final decision from the FDA before the end of the year.

Matt Fellows

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