New Enzyme Replacement Therapy Approved by FDA to Treat Fabry Disease

The Food and Drug Administration (FDA) approved a new treatment for adult patients diagnosed with Fabry disease, sometimes referred to as “Anderson-Fabry disease”. Pegunigalsidase alfa (ELFABRIO; Chiesi Global Rare Diseases, Boston, MA, and Protalix BioTherapeutics, Inc, Carmiel, Israel) represents a unique PEGylated, covalently crosslinked form of α-galactosidase A developed as enzyme replacement therapy (ERT) to treat individuals with Fabry disease. Treatment is administered as an intravenous infusion every 2 weeks.

Data from clinical studies evaluating the drug’s safety and efficacy have demonstrated that pegunigalsidase alfa provided noninferior efficacy in terms of controlling estimated glomerular filtration decline, a marker of kidney disease, compared with treatment with agalsidase beta. Important safety information about treatment with pegunigalsidase alfa includes a black box warning of severe hypersensitivity reactions, including anaphylaxis.

Dror Bashan, Protalix’s President and Chief Executive Officer, stated “The totality of clinical data suggests that ELFABRIO has the potential to be a long-lasting therapy. Together with Chiesi, we are grateful to all of the patients and investigators and their staff members who participated in our clinical trial programs and remain committed to bringing ELFABRIO to patients with Fabry disease.”

On May 5, Chiesi Global Rare Diseases and Protalix BioTheraeutics, Inc reported that the European Commission granted marketing authorization to pegunigalsidase alfa to treat adults with Fabry disease in the European Union. Fabry disease is a rare X-linked genetic condition that affects an estimated 1 in 1000 to 9000 individuals; it is caused by a deficiency in the activity of lysosomal enzyme α-galactosidase A.