Posted by Michael Wonder on 19 Jun 2020
Dicerna receives rare paediatric disease designation from U.S. FDA for nedosiran for the treatment of primary hyperoxaluria
18 June 2020 - Dicerna Pharmaceuticals today announced that the U.S. FDA has granted rare paediatric disease designation for nedosiran, an investigational RNAi therapy being developed as a once-monthly treatment for primary hyperoxaluria.
Primary hyperoxaluria is a family of ultra-rare, life-threatening genetic disorders that cause complications in the kidneys. There are three known types of PH (PH1, PH2 and PH3), each resulting from a mutation in one of three different genes.
Nedosiran is the Company’s lead product candidate and is in development for PH types 1, 2 and 3.
