PhaseRx Receives Positive Opinion for Orphan Drug Designation for PRX-OTC from European Medicines Agency
PHASERX (PZRX)
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Company Research
Source: PR Newswire
SEATTLE, April 25, 2017 /PRNewswire/ -- PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical company developing mRNA treatments for life-threatening inherited liver diseases in children, today announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending orphan medicinal product (orphan drug) designation for PRX-OTC for the treatment of ornithine transcarbamylase deficiency (OTCD). OTCD is a rare genetic disorder characterized by a complete or partial lack of the enzyme ornithine transcarbamylase, an essential urea cycle enzyme that facilitates the break down and removal of ammonia from the body. "This positive opinion recommending orphan drug designation for PRX-OTC in the European Union represents another important step forward towards potentially bringing this novel therapy to patients that suffer from the devastating effects of OTCD," said Robert W. Overell, Ph.D., president and c
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