Protalix BioTherapeutics has received Fast Track designation from the U.S. Food and Drug Administration for pegunigalsidase alfa, or PRX-102, the Company’s plant cell-expressed recombinant, pegylated, cross-linked α-galactosidase-A candidate for the treatment of Fabry disease.  

“We are very pleased that the FDA has recognized the potential for pegunigalsidase alfa to fill an unmet need for Fabry patients,” said Moshe Manor, Protalix’s president and chief executive oficer.  “The data generated in our clinical trials of pegunigalsidase alfa thus far, as well as nonclinical data, as presented to the FDA with Protalix’s application for Fast Track designation, demonstrate that pegunigalsidase alfa has the potential to address an unmet medical need for Fabry patients, such as the prevention of renal failure, improved survivability and a positive impact on quality of life.”

Fabry disease is an X-linked multisystem lysosomal storage disorder caused by the absence or reduction of α-galactosidase-A (α-Gal-A) activity, which is a lysosomal enzyme that catalyzes the hydrolysis of globotriaosylceramide (Gb3) from oligosaccharides, glycoproteins and glycolipids.  The absence or reduction of this enzymatic activity leads to the progressive accumulation of glycolipids, especially Gb3, in capillary endothelial cells, podocytes, tubular cells, glomerular endothelial cells, mesangial cells, interstitial cells, cardiomyocytes, fibroblasts, and neurons.  The accumulation of glycosphingolipids (e.g., Gb3) leads to chronic pain, skin lesions, cardiac, deficiencies, and, in particular, renal involvement.