Author Archives: Marta Figueiredo PhD

PRX-102 at Least as Effective as Fabrazyme, Interim Data Show

One year of treatment with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy (ERT), was at least as effective as Fabrazyme (agalsidase beta) at preventing kidney function decline in adults with Fabry disease, interim data from the BALANCE Phase 3 clinical trial show. These findings add to the growing body…

FDA Rejects Quick Approval of PRX-102, Plant-based ERT for Adults

The U.S. Food and Drug Administration (FDA) has rejected Protalix BioTherapeutics and Chiesi Global Rare Diseases’ request for accelerated approval of PRX-102 (pegunigalsidase alfa) as the first plant-based, every-other-week enzyme replacement therapy (ERT) for adults with Fabry disease. This decision was not related to any concerns with the safety or effectiveness of PRX-102 shown…

Kidney Function Test Alone May Not Effectively Screen for Fabry

Using only an age-dependent threshold of estimated glomerular filtration rate (eGFR) — a marker of kidney function — indicating mild-to-moderate kidney disease is insufficient to effectively screen for undiagnosed Fabry disease patients among the general population, a study shows. The findings suggest that appropriate screening likely wil require evidence…