Chiesi Group Announces Establishment of New Global Rare Diseases Division

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Date: 06/02/2020
  • Specialized division focused on research and product development for rare and ultra-rare diseases highlights Chiesi’s commitment to building a brighter future for patients
  • Chiesi Global Rare Diseases unit will harness the full resources of Chiesi Group to support rare disease communities, with initial focus on lysosomal storage disorders, hematology and ophthalmology


Parma, Feb. 6, 2020 – Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), today announced the formation of Chiesi Global Rare Diseases, a new business unit that will harness the full resources of the Chiesi Group to advance research and new product development for rare and ultra-rare diseases. The unit will be headquartered in Boston, Massachusetts and have an initial focus on research and product development in lysosomal storage disorders, rare haematology and ophthalmology disorders.


“Chiesi has a long history of success in discovering, developing and commercializing innovative therapies to address unmet needs for people living with rare diseases. With the Chiesi Global Rare Diseases unit, we are taking this to an entirely new level – rededicating and strengthening our efforts to support individuals and families affected by rare diseases all around the world,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. “We are officially launching the division in advance of the WORLDSymposium, one of the most important annual meetings focused on rare diseases. During the meeting, we look forward to connecting with many members of the lysosomal storage disorder and broader rare disease communities to outline our hopes and goals for the division and identify opportunities for us to collaborate in research and patient advocacy.” 


Chiesi Group markets treatments for the lysosomal storage disorders alpha‑mannosidosis and nephropathic cystinosis in select markets outside the U.S. The company is also building and advancing a pipeline of innovative therapies for the treatment of LSDs and other rare diseases. In 2018, Chiesi acquired the rights to commercialize, if approved, pegunigalsidase alfa (or PRX-102) in the U.S., which is an investigational therapy currently in development for the potential treatment of Fabry disease. PRX-102 is currently being evaluated in Phase 3 clinical trials and the efficacy and safety of PRX-102 in this indication have neither been reviewed nor approved by the U.S. Food and Drug Administration. In February 2019, Chiesi also joined the Corporate Council of the National Organization for Rare Disorders (NORD).


“We are very encouraged by the response to the establishment of Chiesi Global Rare Diseases from both the advocacy and treatment communities. We are dedicated to making rapid progress in our research and development programs and to being an active partner in opportunities to support patients and families,” said Mr. Chiesi, adding, “There are more than 7,000 rare diseases and for the vast majority there are no treatments available. We are very excited to put Chiesi’s decades of experience in drug development and dedication to patients to work to make a positive difference in the treatment of many rare diseases in the years ahead. The patients are the beginning and the end of our journey.”



About Chiesi Global Rare Diseases

Chiesi Global Rare Diseases is a business unit of the Chiesi Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care.

About Chiesi Group

Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused healthcare group with 85 years of experience in the pharmaceutical industry and a global presence in 29 countries. Chiesi researches, develops, and markets innovative drugs in the respiratory therapeutics, specialist medicine, and rare disease areas. Its R&D organization is headquartered in Parma (Italy), and is integrated with R&D groups in France, the USA, the UK, and Sweden to advance Chiesi's pre-clinical, clinical, and registration programs. Chiesi employs nearly 6,000 people. Chiesi Group is a certified Benefit corporation. For more information, please visit



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