Alnylam files in USA and EU for lumasiran in the treatment of fatal rare disease, PH1

US RNAi therapeutics company Alnylam Pharmaceuticals (Nasdaq: ALNY) has announced the completion of the rolling submission of a New Drug Application (NDA) to the US Food and Drug Administration for lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO), in development for the treatment of primary hyperoxaluria type 1 (PH1).

PH1 is an ultra-rare, life-threatening disease impacting the kidneys and other vital organs; it affects infants, children, and adults, noted Alnylam, whose shares were up 2.5% at $114.94 in early trading this morning, albeit in a rising US market.

In the USA, lumasiran has previously received Pediatric Rare Disease, Orphan Drug and Breakthrough Therapy designations 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.