M2 EQUITYBITES-September 21, 2017-PhaseRx wins US FDA's orphan drug designation for PRX-ASL for argininosuccinate lyase deficiency
Biopharmaceutical company PhaseRx Inc (NASDAQ:PZRX) reported on Wednesday the receipt of orphan drug designation from the US Food and Drug Administration (FDA) for second drug development candidate PRX-ASL for the treatment of argininosuccinate lyase deficiency (ASLD).
M2 PHARMA-September 21, 2017-FDA Grants Orphan Drug Designation to PhaseRx for PRX-ASL for Treatment of Argininosuccinate Lyase
These enzymes are carbamoyl phosphate synthetase 1 (CPS1), ornithine transcarbamylase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase
(ASL), arginase and N-acetylglutamate synthetase.
Increased citrulline indicates a deficiency in either argininosuccinate synthase or argininosuccinate lyase
deficiency: mutational spectrum in Italian patients and identification of a novel ASL pseudogene.
On the other hand, in uricotelic species, this reaction is the only path of endogenous synthesis of Arg, through the catalytic action of argininosuccinate lyase
and argininosuccinate synthetase on citrulline.
Argininosuccinic aciduria (ASAuria; OMIM 207900) is an autosomal recessive inborn error of the urea cycle caused by deficiency of the enzyme argininosuccinate lyase
(ASL; EC 4.