Dicerna Receives Orphan Drug Designation From U.S. Food and Drug Administration for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency
Dicerna Pharmaceuticals, Inc. (DRNA)
Last dicerna pharmaceuticals, inc. earnings: 2/27 04:05 pm
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Source: Business Wire
LEXINGTON, Mass.--(BUSINESS WIRE)--Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Dicerna’s DCR-A1AT for the treatment of alpha-1 antitrypsin (A1AT) deficiency. A1AT deficiency is a genetic disorder that can cause lung and liver disease. Children with the liver manifestations of the disease may present with yellowing of the skin and whites of the eyes (jaundice) and may progress to forming scar tissue in the liver (cirrhosis). Adults with the liver disease may also develop cirrhosis, progress to liver failure or develop a type of liver cancer called hepatocellular carcinoma.1“The FDA’s orphan drug designation is an important milestone in our development plan for DCR-A1AT for alpha-1 antitrypsin deficiency-associated liver disease and underscores the treatme
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