Trial underway for liver disease-causing genetic disorder treatment
The European Commission granted orphan drug designation to Dicerna Pharmaceuticals’ DCR-A1AT for the treatment of congenital alpha-1 antitrypsin deficiency, a genetic disorder that can lead to liver disease in children and adults, according to a press release.
“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,” Ralf Rosskamp, MD, chief medical officer of Dicerna, said in the release. “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.”
DCR-A1AT is a subcutaneously administered RNA interference therapeutic under investigation for treating liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency.
A1AT deficiency can lead to accumulation of mutated A1AT in the liver, which can in turn lead to increased risk for conditions such as cirrhosis, liver failure, and hepatocellular carcinoma, along with risk for lung disease.
Dicerna is conducting a clinical trial investigating the safety and efficacy of DCR-A1AT after beginning enrollment in the fourth quarter of 2019.