EC Authorizes Fabry Disease Treatment

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EC has granted marketing authorization to Chiesi Global Rare Diseases and Protalix BioTherapeutics for PRX-102 (pegunigalsidase alfa).

The European Commission (EC) has granted marketing authorization to Chiesi Global Rare Diseases, a business unit of Chiesi Group, and Protalix BioTherapeutics, for PRX-102 (pegunigalsidase alfa) as a treatment for adult patients with Fabry disease in the European Union (EU).

According to a May 5, 2023 press release, the EC’s authorization was based on the results from a comprehensive clinical development program that assessed PRX-102 in a group of more than 140 patients over a treatment period of 7.5 years. As PRX-102 is a PEGylated enzyme replacement therapy (ERT), the study was performed in both ERT-naïve and ERT-experienced patients, and included a head-to-head that met its primary endpoint, whereby PRX-102 demonstrated non-inferior efficacy to agalsidase beta in controlling kidney 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, in the press release. “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 [EC] 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,” added Diego Ardigò, head of research and development of Global Rare Diseases at the Chiesi Group, in the press release. “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 [EC’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 CEO, in the press release. “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.”

The therapy is not currently approved by the US FDA, but the safety and effectiveness of pegunigalsidase alfa is under review.

Source: Protalix Biotherapuetics