"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 chief executive officer of PhaseRx. "We believe that PRX-OTC, with its first-in-class mechanism of action that can replace the missing protein in these patients, may address this significant unmet medical need."
Applications for orphan designation are initially reviewed by the COMP. Positive opinions are then forwarded to the European Commission, which is responsible for formally granting the orphan designation. Orphan medicinal product designation in the European Union is given to products that are intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; where prevalence of the condition in the EU is less than 5 in 10,000; and where the product represents a significant benefit over existing treatments.
About PRX-OTC for Ornithine Transcarbamylase Deficiency
Ornithine Transcarbamylase (OTC) is an enzyme in the urea cycle, the body's pathway for metabolizing ammonia which results from protein breakdown. Ornithine Transcarbamylase Deficiency (OTCD), a rare liver disorder caused by an inherited single-gene deficiency, results in hyperammonemia (elevated ammonia in the blood), often leading to irreversible neurological impairment, coma and death. PRX-OTC is an intracellular enzyme replacement therapy (i-ERT) designed to replace the missing or defective enzyme inside the cells of patients with OTCD. PRX-OTC has shown therapeutic potential in a preclinical model of OTCD, including lowering of blood ammonia and survival of 100% of treated mice. PRX-OTC has received Orphan Drug Designation by the U.S. Food and Drug Administration.
PhaseRx is a biopharmaceutical company developing a portfolio of mRNA products to correct life-threatening inherited liver diseases in children. PhaseRx's initial product development focus is on urea cycle disorders, a group of rare genetic diseases that generally present before the age of 12 and are characterized by the body's inability to remove ammonia from the blood. The company's i-ERT approach is enabled by its proprietary Hybrid mRNA TechnologyTM platform and is applicable to a significant number of inherited liver diseases. PhaseRx is headquartered in Seattle. For more information, please visit www.phaserx.com.
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SOURCE PhaseRx, Inc.
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