Lumasiran approved in the EU for the treatment of primary hyperoxaluria Type 1 (PH1) in all ages

PH1 is an ultra-rate orphan disease characterised by excessive oxalate production. Lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 mRNA that encodes glycolate oxidase (upstream of the disease-causing defect), is the first approved treatment option for PH1 in the EU

Source:

Biospace Inc.