DCR-A1AT orphan drug to treat alpha-1 antitrypsin deficiency

April 4, 2020 0 By FM

Dicerna Pharmaceuticals said the US FDA has granted orphan drug designation (ODD) to DCR-A1AT for the treatment of alpha-1 antitrypsin (A1AT) deficiency.

A1AT deficiency is a genetic disorder that can cause lung and liver disease.

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. Dicerna expects to dose the first patient in the second half of 2020.

In December 2019, the European Commission granted ODD to DCR-A1AT for the treatment of congenital alpha-1 antitrypsin deficiency based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency.

DCR-A1AT is a subcutaneously administered ribonucleic acid interference (RNAi) therapeutic.