Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce EU Approval of PRX-102 By Investing.com

Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, and Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx^®, today announced that the European Commission (EC) has granted marketing authorization to PRX-102 (pegunigalsidase alfa) in the European Union (EU) 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. “Our deepest gratitude to all patients and patient advocates who have stood shoulder-to-shoulder with clinical researchers, scientists and regulators during the clinical development program, providing the data needed for this approval. I believe this is a vital ingredient in bringing innovation to the real lives of patients and enabling hope and definitive, integrated solutions.”

“We are delighted that the European Commission has approved PRX-102 for the treatment of adult patients with Fabry disease. The EU authorization is a testament to our commitment to deliver innovative therapies and solutions for people affected by rare diseases,” said Diego Ardigò, M.D., Ph.D., head of research and development of Global Rare Diseases at the Chiesi Group. “As a certified B Corp we are committed to ensuring access to PRX-102 to as many people living with Fabry disease as possible and thank those who participated in our extensive clinical research program. It is important to deliver this new treatment option to reduce the burden of this chronic disease on patients, their families, and the healthcare system.”

“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 Chief Executive Officer. “We are proud of this achievement and believe that this approval further validates our science and technology. Based on solid results from our robust clinical programs, PRX-102 has the potential to be widely used for many years to come. Together with Chiesi, we remain committed to meeting the needs of patients with Fabry disease and bringing this new treatment option to market.”

PRX-102 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.

Pegunigalsidase alfa, an investigational new drug product, is currently not approved by the U.S. Food and Drug Administration (FDA). The effectiveness and safety of pegunigalsidase alfa is under review, but has not yet been approved, by the FDA. Prior to FDA review and approval, no conclusions can be drawn on pegunigalsidase alfa’s efficacy and safety profile. When seeking expanded access, treating physicians should consider all possible risks of treatment with pegunigalsidase alfa. Access must be compliant with all applicable federal and state laws and regulations. Investigators should not seek reimbursement for product provided to patients who participate in a government funded insurance program.