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FDA Grants Orphan Drug Designation to PhaseRx for PRX-ASL for Treatment of Argininosuccinate Lyase Deficiency.

M2 PHARMA-September 21, 2017-FDA Grants Orphan Drug Designation to PhaseRx for PRX-ASL for Treatment of Argininosuccinate Lyase Deficiency


- Seattle, Washington-based mRNA treatments specialist PhaseRx, Inc. (NASDAQ: PZRX) has received orphan drug designation from the US Food and Drug Administration for its second drug development candidate, PRX-ASL, for the treatment of argininosuccinate lyase deficiency (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, the company said.

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 including plasma citrulline, argininosuccinic acid and blood ammonia.

Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

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).

The company'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.

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Publication:M2 Pharma
Date:Sep 21, 2017
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