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- The U.S. Food and Drug Administration (FDA) approved Protalix BioTherapeutics (NYSE:PLX) and Chiesi Group's Elfabrio (pegunigalsidase alfa-iwxj) to treat adult patients with Fabry disease.
- Elfabrio — a PEGylated enzyme replacement therapy (ERT) — is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture aimed to provide a long half-life, according to the companies.
- The safety and efficacy of Elfabrio has been studied in a clinical development program in more than 140 patients with up to 7.5 years of follow up treatment, including a head-to-head trial where Elfabrio showed non-inferior efficacy to agalsidase beta, the companies added.
- Last week the Fabry disease therapy was approved in the EU where it is named PRX-102 (pegunigalsidase alfa).
- Fabry disease is an inherited neurological disorder which occurs when an enzyme called alpha-galactosidase-A cannot properly break down lipids. High levels of lipid builds up in the nervous system areas that control involuntary functions, such as breathing and heartbeat, and also in the eyes and kidneys.
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