Dicerna Pharmaceuticals, Inc. announced that the European Commission (EC) has granted orphan drug designation to Dicerna’s DCR-A1AT for the treatment of congenital alpha-1 antitrypsin (A1AT) deficiency based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). A1AT deficiency is a genetic disorder that can cause liver disease in children and adults, leading to complications such as fatigue, jaundice or unintended weight loss. Patients with A1AT deficiency are also at risk of serious or life-threatening conditions such as cirrhosis, liver failure and hepatocellular carcinoma, and may develop lung disease.
“We are gratified to see regulators recognize the urgent need for the development of a safe and effective therapy for treatment of alpha-1 antitrypsin deficiency,” said Ralf Rosskamp, M.D., chief medical officer of Dicerna. “This orphan drug designation from the European Commission acknowledges the needs of this underserved patient population, and we look forward to continuing to investigate DCR-A1AT’s potential in our recently initiated DCR-A1AT development program that includes a Phase 1/2 study now underway in healthy volunteers.”
In June 2019, the Company submitted a clinical trial application to the Swedish Medical Products Agency for DCR-A1AT for the treatment of patients with A1AT deficiency-associated liver disease and began enrolling healthy volunteers in the Phase 1/2 trial of DCR-A1AT (EudraCT number 2019-001999-11) in the fourth quarter of 2019.
The EMA grants orphan medicinal product designation to investigational drugs intended to treat, prevent or diagnose a life-threatening or chronically debilitating disease affecting fewer than five in 10,000 people in the EU and for which no satisfactory treatment is available. Orphan medicinal product designation provides regulatory and financial incentives for companies to develop and market therapies, including market exclusivity, protocol assistance, fee reductions and EU-funded research.