PhaseRx Inc (PZRX) Stock Rallies on Investor Optimism Over Orphan Drug Designation in the European Union


pzrx logoCan you feel the ground moving beneath your feet? Shares of PhaseRx Inc (NASDAQ:PZRX) skyrocket over 70% today, after the drug maker announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) issued a positive opinion on the Orphan Medicinal Product Application for PRX-ASL, for the treatment of argininosuccinate lyase deficiency (ASLD).

To be eligible for orphan medicinal product designation, the Company had to establish medically plausible evidence that PRX-ASL will provide a significant benefit over existing approved therapies in the European Union for the treatment of ASLD.

ASLD 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 neurological impairment, coma and death. PRX-ASL is an intracellular enzyme replacement therapy (i-ERT) designed to replace the missing or defective enzyme in patients with ASLD, thereby correcting the disease. PRX-ASL has shown therapeutic potential in a preclinical model of ASLD, including reduction in the levels of compounds whose elevation are the hallmark of ASLD such as plasma citrulline, argininosuccinic acid (ASA) and blood ammonia.

“This positive opinion recommending orphan drug designation for PRX-ASL in the European Union is a significant step in the development of this product candidate, and follows a similar decision in September by the U.S. Food and Drug Administration,” said Robert W. Overell, Ph.D., president and chief executive officer of PhaseRx. “We are pleased that the Committee for Orphan Medical Products has affirmed our belief in the need for a new therapeutic for this rare liver disorder.”

PhaseRx operates as a biopharmaceutical company that develops a portfolio of mRNA products to correct inherited, life-threatening liver diseases in children. Its product portfolio targets the three urea cycle disorders ornithine transcarbamylase deficiency; argininosuccinate lyase deficiency; and argininosuccinate synthetase deficiency. The company was founded by Robert W. Overell, Patrick S. Stayton, Allan S. Hoffman, Oliver W. Press, and Paul H. Johnson on March 9, 2006 and is headquartered in Seattle, WA.

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