Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for Pegunigalsidase Alfa for Treatment of Fabry Disease

pharmafile | March 3, 2023 | News story | Business Services  

Manchester, UK, 3rd March 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, and Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company, announced today that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending marketing authorisation for PRX-102 (pegunigalsidase alfa), the first and only pegylated enzyme for the treatment of adult patients with Fabry disease.


Chiesi and our partners at Protalix are deeply committed to people living with Fabry disease and their families, many of whom experience unmet medical needs,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. “Our deepest gratitude to all the individuals with Fabry disease who have participated in clinical trials. Thanks to them, PRX-102 has been extensively studied during the clinical development program, providing the data for the CHMP’s evaluation and positive opinion for PRX-102. We look forward to advancing towards approval and launch in Europe and will continue our mission to deliver this potential new treatment option to people living with Fabry disease around the world.”


PRX-102 is a novel recombinant α-Galactosidase-A (α-Gal-A) enzyme being investigated as an enzyme replacement therapy (ERT) for the treatment of Fabry disease. The positive CHMP opinion was based on a marketing authorisation application (MAA) that includes positive data from a comprehensive set of preclinical, clinical and manufacturing studies evaluating PRX-102. The clinical development program includes the completed Phase 3 BALANCE, BRIDGE, and BRIGHT clinical trials, the Phase 1/2 clinical trial, and ongoing related extension studies. PRX-102 has been studied in more than 140 patients, consisting of both ERT-naïve and ERT-experienced patients, and includes a head-to-head trial versus agalsidase beta.

Professor Derralynn Hughes, Professor of Experimental Haematology, University College London, and Principal Investigator for BALANCE, BRIDGE, and BRIGHT, commented: “Fabry is a rare disease with high unmet needs. People living with Fabry disease experience a significant symptom and treatment burden that can have a serious and debilitating impact on their overall quality of life. The CHMP positive opinion is welcome news for the Fabry community and, if approved, will offer an important new option for treating this debilitating and complex disease.” 

The CHMP opinion is now referred for final action to the European Commission (EC). A final EC decision on the MAA is expected in the beginning of May 2023.



About Fabry Disease

It has been estimated that 1,350 people in the UK have symptomatic Fabry disease, a rare, progressive, X-linked inherited lysosomal storage disorder, caused by a genetic mutation which leads to a deficiency of enzyme α-galactosidase A. This deficiency leads to progressive build-up of globotriaosylceramide (Gb3 or GL3) and globotriaosylsphingosine (lyso-Gb3) in the plasma and lysosomes of cells throughout the body. This accumulation causes metabolic dysfunction and cell death, leading to progressive organ failure. Fabry disease affects multiple organ systems, and may lead to renal dysfunction, cardiac manifestations, neuropathic pain, cerebrovascular disease, gastrointestinal symptoms, angiokeratomas and hypohidrosis/anhidrosis. Adults with Fabry disease may be treated by intravenous infusion with enzyme replacement therapy (ERT) to replace the function of the defective α-galactosidase A enzyme.  


About Pegunigalsidase Alfa (PRX–102)

Pegunigalsidase alfa (PRX–102) is an investigational, plant cell culture-expressed, and chemically modified version of the recombinant a-galactosidase A enzyme.  Protein subunits are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with a mean circulatory half-life of approximately 80 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 please visit: 


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 companywide 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 at becoming net-zero by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 30 countries, and counts more than 6,000 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.


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