Chiesi Global Rare Diseases Announces Publication of Results from Phase 3 BALANCE Study in Fabry Disease

- Data from the first randomised, double-blind, active-control, head-to-head clinical trial of enzyme replacement therapies (ERTs) in Fabry disease published in the Journal of Medical Genetics -

PARMA, January 10, 2024 - 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 the publication of data from the Phase 3 BALANCE study that evaluated the efficacy, safety and tolerability of pegunigalsidase alfa versus agalsidase beta in adult patients with previous agalsidase beta treatment and deteriorating renal function. The results are published in the peer-reviewed Journal of Medical Genetics (“Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study” Wallace EL, et al. J Med Genet 2023; doi: 10.1136/jmg-2023-109445).

Pegunigalsidase alfa, a PEGylated enzyme replacement therapy (ERT), is a recombinant human α-Galactosidase A enzyme expressed in plant-cell culture. It is now approved for the treatment of adults with Fabry disease in the United States, European Union, and Great Britain.

“There continues to be a general lack of awareness and a limited appreciation of the numerous unique challenges that people living with Fabry disease face,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases. “This is the first randomized, double-blind, head-to-head clinical trial of ERTs in Fabry disease, and it would not have been possible without the incredible support from investigators, patients, and families who dedicated two years of their lives to advance this important research. We are pleased to provide patients with Fabry disease an additional treatment option and will continue our work to support millions of people affected by rare diseases around the world.”

The BALANCE study included 77 adults with Fabry disease with an annualized eGFR slope more negative than −2mL/min/1.73m²/year who had received agalsidase beta for at least one year (on average almost six years). Participants were randomized 2:1 to receive 1mg/kg of pegunigalsidase alfa or agalsidase beta every two weeks for two years.

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 Contacts

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
Tel: +1 212 253 8881
Email: adaley@berrypr.com