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."
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.
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 devastating consequences, including cumulative and irreversible neurological impairment, coma and death. The only known cure for ASLD is a liver transplant. Currently available drug treatments do not correct the disease, and do not eliminate the risk of life-threatening crises.
PhaseRx is a biopharmaceutical company dedicated to developing mRNA products for the treatment of children with inherited enzyme deficiencies in the liver using intracellular enzyme replacement therapy (i-ERT). PhaseRx's initial product development focus is on urea cycle disorders, a group of rare genetic diseases that generally present before the age of twelve and are characterized by the body's inability to remove ammonia from the blood with potentially devastating consequences for patients. The company's i-ERT approach is enabled by its proprietary Hybrid mRNA Technology™ platform. PhaseRx is headquartered in Seattle. For more information, please visit www.phaserx.com.
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SOURCE PhaseRx, Inc.
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