PhaseRx Inc (NASDAQ:PZRX) reported that the European Medicines Agency’s COMP has given a positive view recommending orphan medicinal product status for PRX-ASL, for the cure of argininosuccinate lyase deficiency, the company’s second drug candidate to cure a urea cycle disorder.
ASLD can be stated as a rare liver disorder, which is a result of an inherited single-gene deficiency leading in hyperammonemia. It can even result in irreversible neurological impairment, death and coma. PRX-ASL is an i-ERT intended to replace the defective or missing enzyme in people with ASLD, thereby correcting the ailment. PRX-ASL has demonstrated therapeutic prospect in a preclinical model of ASLD, counting decline in the compounds levels whose elevation are the mark of ASLD such as argininosuccinic acid, blood ammonia and plasma citrulline.
Robert W. Overell, Ph.D., the CEO and President of PhaseRx, expressed that this positive stance recommending orphan drug status for PRX-ASL in the EU is a notable step in the advancement of this candidate, and follows a same decision by the U.S. FDA in September. They are delighted that the respective committee has affirmed their belief in the requirement for a new therapeutic for this disease. Applications for orphan designation are primarily reviewed by the COMP. Constructive opinions are then sent to the European Commission, which is accountable for formally allowing the orphan designation.
This status in the European Union is provided to products that are planned for the treatment, diagnosis or prevention of a disease that is chronically debilitating or life-threatening; where prevalence of the disease in the European Union is less than five in 10,000; and where the offering exhibits a notable benefit over current treatments. PhaseRx is a biopharmaceutical firm committed to advancing mRNA offerings for the treatment of kids with hereditary enzyme deficiencies in the liver utilizing intracellular enzyme replacement therapy.