Pegunigalsidase Alfa Stabilized Glomerular Filtration in Fabry Disease

In a phase 3 clinical trial (NCT03180840), participants treated with pegunigalsidase alfa (PRX 102; Protalix BioTherapeutics, Carmiel, Israel; and Chiesi Global Rare Diseases, Boston, MA) for Fabry disease had stabilization of their estimated glomerular filtration rate (eGFR) values.
Mean change in eGFR from baseline was 1.27 mL/min/1.73 m2(1.39). At the end of the study, the mean (SE) eGFR slope for the overall population was 2.92 (1.05) mL/min/1.73m2/year, indicating stability.

"We are excited to share the final data from the BRIGHT study, an important milestone in the progress of our PRX-102 clinical program," said Dror Bashan, president and chief executive officer, Protalix. "The availability of this data for review by the US 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 adults with Fabry disease." 

Pegunigalsidase alfa is a plant cell-expressed recombinant, PEGylated, cross-linked α galactosidase A product candidate test in the BRIGHT trial, which was a switch-over study 
in which treatment of 2mg/kg was administered every 4 weeks for 52 weeks. Participants (24 men and 6 women) had a mean age of 40.5 (range 19 to 58) and previously received an approved enzyme replacement therapy (ERT) for at least 3 years. The most common Fabry disease symptoms at baseline were acroparesthesia, heat intolerance, angiokeratomas, and hypohydrosis. 

Treatment-emergent adverse events (TEAEs), primarily infusion-related reactions) occurred in 9 (30.0%) participants, and all were mild or moderate in severity, with most resolving by the end of the study. There were no serious or severe treatment-related TEAEs, and no TEAEs led to death or study withdrawal.