Posted by Michael Wonder on 16 Jul 2019
Dicerna receives breakthrough therapy designation for DCR-PHXC for treatment of primary hyperoxaluria type 1
15 July 2019 - FDA recognises primary hyperoxaluria Types 2 and 3 as meeting criteria for a serious or life-threatening disease or condition.
Dicerna Pharmaceuticals today announced that the U.S. FDA has granted a breakthrough therapy designation to DCR-PHXC for the treatment of patients with primary hyperoxaluria type 1.
DCR-PHXC is the only RNAi investigational therapy in development for the treatment of all types of PH, a family of severe, rare, inherited disorders of the liver that often result in kidney failure.
