CHMP recommends Alnylam's RNAi therapeutic for approval

By The Science Advisory Board staff writers

October 16, 2020 -- The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) posted a positive opinion recommending approval for Alnylam Pharmaceutical's lumasiran, an investigational RNA interference (RNAi) therapeutic for the treatment of primary hyperoxaluria type 1 (PH1).

Lumasiran targets hydroxyacid oxidase 1 (HAO1) messenger RNA (mRNA), which encodes glycolate oxidase. The RNAi therapeutic mediates RNAi, or the cellular process of gene silencing, by potently silencing mRNA that encodes disease-causing or disease pathway proteins.

CHMP rendered a positive opinion on lumasiran. If approved by the European Commission, the drug will be marketed in Europe under the brand name Oxlumo.

The positive opinion is based on efficacy and safety findings in two clinical trials. In both studies, endpoint analysis demonstrated the reduction of urinary and plasma oxalate, and the normalization or near-normalization of urinary oxalate in response to the RNAi therapeutic.

PH1 is a rare orphan disease that causes excessive oxalate production, which can result in end-stage renal disease. It impacts 3.5 to 4 individuals per 1 million in Europe and the U.S.

The U.S. Food and Drug Administration has granted a priority review for the new drug application submitted by the company and has set an action date of December 3, 2020.

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