EU clears Protalix, Chiesi's enzyme replacement therapy for Fabry disease

2023-05-05

临床结果临床3期上市批准

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.

Sanofi reported earlier this year that Fabrazyme generated sales of €938 million ($1 billion) in 2022.

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机构

Protalix Ltd.