EU clears Protalix, Chiesi's enzyme replacement therapy for Fabry disease
05 May 2023
Clinical ResultPhase 3Drug Approval
Protalix BioTherapeutics and partner Chiesi Farmaceutici announced Friday that the European Commission has authorised PRX-102 (pegunigalsidase alfa) to treat adults with Fabry disease. The PEGylated enzyme replacement therapy (ERT) is a recombinant human α-Gal-A enzyme expressed in plant-cell culture that is designed to provide a long half-life.
Dror Bashan, chief executive at Protalix, called the decision a "significant milestone" for patients. "Based on solid results from our robust clinical programmes, PRX-102 has the potential to be widely used for many years to come," he added. The European Medicines Agency's (EMA) drug advisory body adopted a positive opinion for the filing back in February.
According to the companies, the EU nod is based on results from a clinical development programme involving more than 140 patients with up to 7.5 years of treatment. PRX-102 has been studied in both ERT-naïve and ERT-experienced patients, including the Phase III head-to-head BALANCE trial where it met its primary endpoint of non-inferiority against Sanofi's Fabrazyme (agalsidase beta) at controlling kidney disease based on estimated glomerular filtration rate (eGFR) decline.
PRX-102 is not currently approved in the US, where in 2021 the FDA issued a complete response letter for the product. The companies resubmitted their application late last year with what they said was a "comprehensive set of clinical and manufacturing data," including data from all three Phase III studies – BALANCE, BRIDGE and BRIGHT – as well as safety data compiled from the Phase III extension studies of PRX–102.
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