RARE Daily

European Commission Approves Chiesi’s and Protalix’ Fabry Disease Treatment

May 5, 2023

Rare Daily Staff

The European Commission granted marketing authorization to Chiesi Global Rare Diseases and Protalix Therapeutics’ PRX-102 for the treatment of adult patients with Fabry disease.

“People living with Fabry disease often perceive their disease as burdensome and still experience unmet medical needs,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases, a business unit of Chiesi Group.

Fabry disease is an X–linked inherited disease that results from deficient activity of the lysosomal α–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in the lysosomes throughout a person’s body. Fabry disease occurs in one person per 40,000 to 60,000 people. Fabry patients inherit a deficiency of the α–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time and, accordingly, Gb3 accumulates, primarily in the blood vessel and tissues. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure.

PRX-102 (pegunigalsidase alfa) is a PEGylated enzyme replacement therapy (ERT). It is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life.

The EC authorization of PRX-102 is based on results from a comprehensive clinical development program in more than 140 patients with up to 7.5 years of treatment. It has been studied in both ERT-naïve and ERT-experienced patients, including a head-to-head trial that met its primary endpoint, with PRX-102 demonstrating non-inferior efficacy to agalsidase beta in controlling kidney disease as evaluated by the estimated glomerular filtration rate (eGFR) decline.

“The European Commission’s approval of PRX-102 is a significant milestone for patients with Fabry disease and their families, providing a new therapeutic option,” said Dror Bashan, Protalix’s president and CEO.

Pegunigalsidase alfa, is currently under review, but has not yet been approved, by the U.S. Food and Drug Administration.

Photo: Dror Bashan, president and CEO of Protalix

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