Replies to post #3643 on PROTALIX BIOTHERAPEUTICS, INC. (PLX)

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Protalix, Chiesi report final data from late-stage study of PRX-102 for Fabry disease
Mar. 18, 2022 7:46 AM ETProtalix BioTherapeutics, Inc. (PLX)

By: Ravikash, SA News Editor1 Comment

Protalix BioTherapeutics (NYSE:PLX) and Chiesi Global Rare Diseases, a unit of Chiesi Farmaceutici, reported final results of a phase 3 trial evaluating pegunigalsidase alfa (PRX-102) to treat Fabry disease.

The company said data from the 52-week study, dubbed BRIGHT, suggested that treatment with 2 mg/kg of PRX-102 administered by intravenous infusion every four weeks was well tolerated, and Fabry disease assessed by estimated glomerular filtration rate and plasma lyso-Gb3 concentration was stable.

"The availability of this data for review by the U.S. Food and Drug Administration, the European Medicines Agency and other regulators is another step forward towards the anticipated approval of PRX-102 as a potential good alternative for adult Fabry patients in both the regular 1 mg\kg every two weeks as well as the 2 mg\kg every four weeks regimen," said Protalix's President and CEO Dror Bashan.

The trial enrolled 24 males and 6 females who previously received an approved enzyme replacement therapy (agalsidase alfa – Replagal or agalsidase beta – Fabrazyme) for at least three years.

The company said overall, 33 of 182 total treatment-emergent adverse events reported in nine (30%) patients were considered treatment-related. All were mild or moderate in severity and the majority were resolved at the end of the study.

In February, the companies filed for EU approval of PRX-102 for Fabry disease — an inherited disorder that results from deficient activity of the lysosomal a–Galactosidase–A enzyme causing progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide in blood vessel walls throughout the body.