Alnylam Pharmaceuticals initiates a rolling submission to the FDA for lumasiran for the treatment of primary hyperoxaluria type 1 .

Alnylam Pharmaceuticals, Inc. has initiated a rolling submission of its New Drug Application (NDA) to the FDA for lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria type 1 (PH1). The rolling submission allows completed sections of an NDA to be reviewed by the FDA on an ongoing basis. Specifically, Alnylam has submitted the non-clinical components to the FDA and expects to submit the remaining components in early 2020. Alnylam also announced that it has been granted a pediatric rare disease designation from the FDA for lumasiran for the treatment of PH1. Comment; Lumasiran has also received both U.S. and EU Orphan Drug Designations, a Breakthrough Therapy Designation from the FDA and a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). Alnylam intends to file a Marketing Authorisation Application (MAA) with the EMA in early 2020.