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Chiesi Group Announces Establishment of New Global Rare Diseases Division

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 commercialising 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 (LSDs) 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, a long-term enzyme replacement therapy in the U.S., which is an investigational therapy currently in development for the potential treatment of Fabry disease. The therapy is currently being evaluated in Phase 3 clinical trials and the efficacy and safety of the therapy in this indication have neither been reviewed nor approved by the U.S. Food and Drug Administration.

“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.”

Tom Delahoyde, Managing Director of Chiesi in the UK and Ireland, said, “This is an exciting step to helping the millions of people affected by rare and ultra-rare diseases whose treatment options are often limited. The new unit will enable us to focus our efforts on providing people with rare conditions with the prospect of improved lives and evidence-based treatment. We will be working closely with our colleagues in the US with the aim of bringing advance research and new products in this area to the UK and Ireland.“