Israeli drug developer Protalix Biotherapeutics Inc. (NYSE:PLX; TASE: PLX) announced today that the US Food and Drug Administration (FDA) has granted Fast Track designation to pegunigalsidase alfa (PRX-102), the company’s plant cell-expressed recombinant, pegylated, cross-linked α-galactosidase-A candidate for the treatment of Fabry disease. The FDA’s Fast Track designation is for treatments for serious conditions that fill an unmet medical need.
Protalix president and CEO Moshe Manor said, “We are very pleased that the FDA has recognized the potential for pegunigalsidase alfa to fill an unmet need for Fabry patients. 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. “We believe that Fast Track designation will help facilitate our development program for pegunigalsidase alfa and may shorten the timelines to an anticipated approval, which will greatly benefit Fabry patients.”
Fabry disease is a serious, life-threatening condition. It is a disease or condition associated with morbidity that has a substantial impact on survival, day-to-day function, and the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one. 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. End-stage renal failure and cardiomyopathy often lead to early death in Fabry patients. Fabry disease causes substantial reduction in life-expectancy, by an average of 15 years in female patients and 20 years in male patients, compared to the general population.
Pegunigalsidase alfa is currently being studied globally in three phase III clinical trials. Enrollment in each of the trials continues to progress and estimated timelines for top-line data announcements will be announced upon completion of enrollment for each individual trial.
Published by Globes [online], Israel business news - www.globes-online.com - on January 31, 2018
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