Posted by Michael Wonder on 08 Apr 2020
Alnylam completes rolling submission of new drug application to the U.S. FDA and submits marketing authorisation application to the EMA for lumasiran for the treatment of primary hyperoxaluria type 1
7 April 2020 - Lumasiran is the first potential therapeutic to demonstrate substantial reduction in urinary oxalate excretion.
Alnylam Pharmaceuticals today announced the completion of the rolling submission of a new drug application to the U.S. FDA for lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase, in development for the treatment of primary hyperoxaluria type 1 (PH1).
In the U.S, lumasiran has previously received paediatric rare disease designation, orphan drug designation, and breakthrough therapy designation for the treatment of PH1, based on data showing a substantial reduction in urinary oxalate, the key toxic metabolite responsible for the clinical manifestations of the disease.
