PH1 is a severe, rare, inherited disorder of the liver that often results in kidney failure and for which there are no approved therapies.
Orphan Drug Designation by the EMA provides regulatory and financial incentives under Regulation (EC) No. 141/2000 for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and for which no satisfactory treatment is available.
In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to the centralized marketing authorization procedure.
DCR-PH1 is Dicerna’s proprietary therapeutic candidate in development for the treatment of PH1. Preclinical experiments indicate that DCR-PH1 knocks down hydroxyacid oxalyase-1 (HAO1), the gene transcript that encodes for the enzyme glycolate oxidase (GO), which, in turn, reduces the excretion of oxalate in the urine.
"We are very pleased to have received Orphan Drug Designation in the EU for DCR-PH1. This is an important regulatory milestone for our team working to bring this therapy to patients with PH1," said Ted Ashburn, M.D., Ph.D., senior vice president of product strategy and operations at Dicerna.
"We are encouraged by the progress of this program to date and our aim is to rapidly advance the development of DCR-PH1 as a potential new treatment option that will have a meaningful impact on reducing the disease burden for PH1 patients."
DCR-PH1 incorporates a proprietary, lipid nanoparticle (LNP) technology that allows for efficient delivery to the liver after intravenous (IV) administration. Dicerna obtained rights to this delivery technology by way of a licensing agreement with Tekmira Pharmaceuticals Corporation signed in November 2014.