PhaseRx announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation for PRX-OTC for the treatment of ornithine transcarbamylase deficiency (OTCD). 

OTCD is caused by an inherited single-gene deficiency and results in hyperammonemia, which can lead to irreversible neurologic impairment, coma, and death. For patients with OTCD, the only cure is a liver transplant. Pharmacologic treatments currently on the market neither correct the disease nor eliminate the risk of life-threatening crises. 

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PRX-OTC, an intracellular enzyme replacement therapy, is designed to replace the missing or defective enzyme in patients with OTCD to correct the disease. The drug candidate utilizes the Company’s proprietary Hybrid mRNA Technology platform that results in synthesis of intended proteins in hepatocytes with a fast onset of action. This suggests a highly effective delivery of mRNA molecules and the synthesis of a number of protein classes including cytosolic proteins, mitochondrial proteins and secreted proteins. In a preclinical model, PRX-OTC demonstrated therapeutic potential with 100% survival in treated mice and a decrease in blood ammonia. 

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