|Protalix BioTherapeutics Receives Positive Opinion for Orphan Designation for PRX-102 for the Treatment of Fabry Disease in the European Union|
pegunigalsidase alfa is the Company's PEGylated, chemically-modified version of the recombinant alpha-Galactosidase-A protein, for the treatment of Fabry disease. pegunigalsidase alfa is currently being evaluated globally in three phase III clinical trials.
To be designated an Orphan Medicinal Product, the sponsor must establish that no satisfactory method of diagnosis, prevention or treatment of the condition can be authorized in the
“We are delighted to receive the positive opinion for orphan drug designation for PRX-102 for the treatment of Fabry disease. The action from the EMA on this decision mirrors our own strategy for developing PRX-102 as a potential better therapeutic alternative for people living with Fabry disease, a patient population in need of new treatment options,” said Mr.
Orphan Medicinal Product designation potentially qualifies the sponsor for 10 years of marketing exclusivity upon approval, as well as certain fee reductions. The sponsor will also be eligible for access to a centralized authorization procedure, including applications for marketing authorization and inspections, and for protocol assistance.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix’s unique expression system presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner. Protalix’s first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the U.S. Food and Drug Administration (
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