CHMP Issues Positive Opinion on Chiesi and Protalix’s Fabry Disease ERT

Rare Daily Staff

European Medicines Agency’s Committee for Medicinal Products for Human Use recommended marketing authorization for Chiesi Global Rare Diseases and Protalix BioTherapeutics’ PRX–102, the first and only pegylated enzyme for the treatment of adult patients with Fabry disease.

The CHMP opinion is now referred for final action to the European Commission. A final EC decision on the application is expected in the beginning of May 2023.

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 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 – particularly of the kidneys, but also of the heart and the cerebrovascular system.

PRX–102 is an experimental, novel, PEGylated enzyme replacement therapy under development to treat unmet medical needs for Fabry patients, such as progressive kidney decline. PRX-102 is a plant cell culture-expressed, and chemically modified stabilized version of the recombinant α–Galactosidase–A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours.

“We believe that this recommendation further recognizes the strength of the positive dataset from our robust clinical trial program and underscores the potential for PRX–102 to provide a new treatment option for patients with Fabry disease,” said Dror Bashan, Protalix’s president and CEO. “Data from our clinical program indicates that PRX–102 has the potential to be a long-lasting therapy with a favorable tolerability and immunogenicity profile.”

Photo: Dror Bashan, Protalix’s president and CEO