Chiesi Global Rare Diseases Announces European Commission (EC) Authorization of PRX-102 (pegunigalsidase alfa) for the Treatment of Fabry Disease

Pegylated enzyme replacement therapy designed to provide a long half-life*

Updated August 17, 2023

PARMA, Italy and BOSTON, May 5, 2023 - Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, 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.”

PRX-102, a PEGylated enzyme replacement therapy (ERT), is a recombinant human alpha-galactosidase A enzyme expressed in plant-cell culture that is designed to provide a prolonged 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 follow up treatment. PRX-102 demonstrated consistent and robust efficacy findings in treatment-naïve patients, including a significant reduction in renal Gb3 inclusions in the peritubular capillaries (PTC) as well as a positive effect on the rate of loss of renal function (as measured by annualized change in estimated eGFR) in the overall clinical program. PRX-102 has been studied in a head-to-head trial vs agalsidase beta. PRX-102 was generally well-tolerated with the majority of adverse events being mild or moderate in severity.

*The pharmacokinetic results demonstrated that the enzyme was available throughout the 2-week intervals with a plasma half-life ranging from 53-134 hours across dose groups and visit day. Clinical studies have not established that pharmacological characteristics, including half-life, result in superior efficacy or safety based on clinically relevant endpoints.

About Fabry Disease
Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal alpha-galactosidase A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb₃) in the lysosomes throughout a person’s body. Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the alpha-galactosidase A enzyme, which is normally responsible for the breakdown of Gb₃. The abnormal storage of Gb₃ increases with time and, accordingly, Gb₃ accumulates, primarily in the blood vessel and tissues. The ultimate consequences of Gb₃ deposition range from episodes of pain and impaired peripheral sensation to end-organ failure.

About PRX-102
PRX102 (pegunigalsidase alfa), a PEGylated enzyme replacement therapy (ERT) to treat Fabry disease, is a plant cell culture-expressed, and chemically modified stabilized recombinant version of the alpha-galactosidase A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with stable pharmacokinetic parameters. In clinical studies, PRX102 has been observed to have an initial half-life of 78.9 ± 10.3 hours.

About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.
For more information visit www.chiesirarediseases.com.

About Chiesi Group
Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.

By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we’re part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.

With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 31 countries, and counts more than 6,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.
For further information please visit www.chiesi.com.

Chiesi Group Media Contact
Chiara Travagin
Rare Communication Manager
Tel: +39 348 8818985
Email c.travagin@chiesi.com

Alessio Pappagallo
Press Office Manager
Tel: +39 339 5897483
Email a.pappagallo@chiesi.com

Adam Daley
Berry & Company Public Relations
1-212-253-8881
adaley@berrypr.com