Chiesi creates Boston rare disease unit ahead of Fabry launch

Chiesi Farmaceutici has set up a rare disease unit in Boston. The division will develop treatments for patients with rare lysosomal storage, hematological and ophthalmological disorders.

Italy-based Chiesi has built up a portfolio of rare disease assets and prospects in recent years. Chiesi already sells drugs against lysosomal storage disorders (LSDs) alpha‑mannosidosis and nephropathic cystinosis outside the U.S. In 2018, Chiesi positioned itself to expand its rare disease activities into the U.S. by picking up the rights to Protalix BioTherapeutics’ Fabry disease drug in the country.

The creation of a global rare diseases unit based out of Boston is the next step in the process. Chiesi sees the unit as a way to support development of rare disease drugs, with an initial focus on LSDs, hematology and ophthalmology.

Following the rare disease biotech playbook, Chiesi plans to connect with communities of patients who have the diseases it hopes to treat. Operating out of the U.S., where many rare disease groups are based, and specifically the biotech hot spot of Boston could further Chiesi’s dual ambitions for the unit in patient advocacy and R&D.

Giacomo Chiesi, the former head of global corporate development at Chiesi, is serving as head of the rare disease division. Other Boston-based rare disease staff, including ex-Rhythm Pharmaceuticals Vice President Lawrence Bressler, are already in place.

Chiesi is in the process of taking on more staff in Boston. The Italian drugmaker lists nine open posts in the city, including the positions of senior director, U.S. market access and global head of patient advocacy for its rare disease business unit. Chiesi is looking to take on people with those skills ahead of the anticipated launch of its Fabry candidate PRX-102. 

Protalix granted Chiesi the U.S. rights to the drug for $20 million upfront in 2018. Since then, the candidate has advanced to the point that Chiesi and Protalix are gearing up to file for FDA approval in April. Winning FDA approval in Fabry disease will put Chiesi in competition with Sanofi’s Genzyme unit, arguably the pioneer of the rare disease model it now hopes to replicate.