PhaseRx (PZRX) Receives Orphan Drug Designation for Treatment of Ornithine Transcarbamylase Deficiency
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PhaseRx Receives Orphan Drug Designation from FDA for PRX-OTC for the Treatment of Ornithine Transcarbamylase Deficiency
November 28, 2016 8:30 AM ESTSEATTLE, Nov. 28, 2016 /PRNewswire/ -- PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical company developing mRNA treatments for life-threatening inherited liver diseases in children, today announced that its lead candidate, PRX-OTC, which is being developed for the treatment of ornithine transcarbamylase deficiency (OTCD), has received orphan drug designation by the U.S. Food and Drug Administration (FDA).
OTCD is a rare liver disorder caused by an inherited single-gene deficiency that results in hyperammonemia (elevated ammonia in the blood), and can lead to irreversible... More
