Research & Development
US FDA grants breakthrough Therapy Designation to Dicerna Pharmaceuticals' DCR-PHXC
17 July 2019 -

The US Food and Drug Administration (FDA) has granted breakthrough Therapy Designation to United States-based Dicerna Pharmaceuticals' DCR-PHXC intended for the treatment of patients with primary hyperoxaluria (PH) type 1, it was reported yesterday.

The product 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.

In its communication to the company, the US FDA also conveyed its determination that PH type 2 (PH2) and PH type 3 (PH3) meet the criteria for a serious or life-threatening disease or condition, based on the Agency's standards. The firm will continue its ongoing dialogue with the US FDA regarding endpoints for studies of DCR-PHXC in patients with PH2 and PH3, as part of the PHYOX clinical development program.