Enzyme Replacement Therapy OK’d for Fabry Disease

FDA has approved a Chiesi Global Rare Diseases and Protalix BioTherapeutics BLA for Elfabrio (pegunigalsidase alfa-iwxj) for treating adult patients with Fabry disease. The PEGylated enzyme replacement therapy (ERT) contains the recombinant human α–Galactosidase-A enzyme, which is expressed in a plant-cell culture that is designed to provide a long half-life, the companies say.

Approval was based on data in more than 140 patients with up to 7.5 years of follow up treatment. “It has been studied in both ERT-naïve and ERT-experienced patients,” the companies say, “including a head-to-head trial that met its primary endpoint with Elfabrio demonstrating non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline, and in which Elfabrio was generally well-tolerated with the majority of adverse events being mild or moderate in severity.”