Dicerna Receives Orphan Drug Designation for PH1 Treatment

Dicerna Pharmaceuticals announced on April 23, 2015 that it received FDA Orphan Drug designation for its primary hyperoxaluria type 1 (PH1) treatment, DCR-PH1. FDA grants Orphan Drug designation to drugs that are intended for the treatment of diseases or disorders that affect fewer than 200,000 people in the United States, or that affect more than 200,000 persons but that will not recover the costs of developing and marketing the drug.

There are no approved therapies for PH1, which is a severe, rare, inherited disorder that often results in kidney failure. DCR-PH1 is a lipid nanoparticle technology that can be efficiently delivered to the liver via an intravenous administration. According to a press release, the treatment is in preclinical development and is engineered to “address the pathology of PH1 by targeting and destroying the messenger RNA (mRNA) produced by HAO1, a gene implicated in the pathogenesis of PH1. HAO1 encodes glycolate oxidase, a protein involved in producing oxalate. By reducing oxalate production, this approach seeks to prevent the complications of PH1.”

Source: Dicerna Pharmaceuticals