Dicerna's (DRNA) A1AT Candidate Gets Orphan Drug Tag In EU
Zacks Investment Research | Dec 17, 2019 11:03PM ET
Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA) announced that the European Commission (EC) has granted orphan drug designation to its RNAi therapeutic DCR-A1AT, currently being developed for treating congenital alpha-1 antitrypsin (A1AT) deficiency, a protein primarily produced in and released from the liver.
The prestigious tag for DCR-A1AT was based on a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). Currently, there are no approved therapies to treat A1AT deficiency-associated liver disease.
The EMA usually grants orphan medicinal product status to drugs that target serious or life-threatening conditions, which are being developed to treat, prevent or diagnose diseases or conditions affecting not more than five in 10,000 people in the region. In addition to providing Dicerna with certain benefits and incentives, this status will allow DCR-A1AT to enjoy a period of market exclusivity in the EU, if approved, for the treatment of A1AT deficiency.
Dicerna submitted a clinical trial application to the Swedish Medical Products Agency for DCR-A1AT to treat patients with A1AT deficiency-associated liver disease in June 2019. The company began enrolling healthy volunteers during the fourth quarter in the phase I/II study on DCR-A1AT.
Notably, DCR-A1AT is being evaluated in a phase I/II study for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency.
Shares of Dicerna have skyrocketed 146.5% so far this year, significantly outperforming the Zacks Investment Research
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