PhaseRx leaps on news of FDA orphan status

29 November 2016

US drug developer PhaseRx (Nasdaq: PZRX) saw its shares leap 106.7% to $2.34 after it revealed it had been awarded orphan status for its lead candidate in the USA.

PRX-OTC, which is being developed for the treatment of ornithine transcarbamylase deficiency (OTCD), has received orphan drug designation by the US Food and Drug Administration.

OTCD 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-OTC is an intracellular enzyme replacement therapy (i-ERT) designed to replace the missing or defective enzyme in patients with OTCD, thereby correcting the disease. PRX-OTC has shown therapeutic potential in a preclinical model of OTCD, including lowering of blood ammonia and survival of 100% of treated mice.

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