BRIEF—Breakthrough Therapy award for novel RNAi candidate

USA-based RNAi specialist Dicerna Pharmaceuticals has been given Breakthrough Therapy Designation (BTD) for DCR-PHXC, in primary hyperoxaluria type 1 (PH1).

It is the only RNAi investigational therapy in development for the treatment of all types of PH, a family of severe, rare disorders of the liver.

Chief medical officer Ralf Rosskamp said the award recognizes: “both the urgent need to develop a therapy for primary hyperoxaluria type 1 and the encouraging preliminary data from the PHYOX1 clinical trial of DCR-PHXC in these patients.”