RESEARCH TRIANGLE PARK – Adults living with cystic fibrosis (CF) now have access to a new revolutionary therapy aimed at improving pulmonary function.

Chiesi USA (pronounced (key-A-zee), with U.S. headquarters in Cary, has launched its Bronchitol (mannitol) inhalation powder following approval by the U.S. Food and Drug Administration.

“This is the first approved mucoactive agent for CF patients in nearly 30 years,” Jon Zwinski, Chiesi USA CEO and general manager, told the North Carolina Biotechnology Center.

“It is the only dry-powder inhaled mucoactive agent that is compact, discreet and portable, and does not require any nebulization, refrigeration or routine cleaning.”

CF is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. More than 30,000 people in the U.S. are living with CF (more than 70,000 worldwide).

There’s currently no cure.

In the LimeLight: Cary drug firm Chiesi USA names successor to longtime CEO

This new therapy is billed as an “add-on maintenance therapy” for patients 18 years of age and older, and Zwinski said it could make a big difference with symptoms.

Data from a global Phase 3 study evaluating the efficacy and safety of inhaled dry powder mannitol in adults with CF has been published online in the Journal of Cystic Fibrosis.

In the multicenter, double-blind, randomized, parallel-group, controlled study of 423 adults with CF, subjects received either mannitol 400mg or mannitol 50mg (the control), twice daily via dry powder inhaler for 26 weeks. Authors concluded that the 400mg dose “significantly improved” lung function compared with the control group.

“Thanks to evolving treatments over the years, many patients are now living longer, more active lives,” Zwinski said.

“Until a cure is found, it is critical to continue to provide options to patients so they can find the right treatment that fits their needs – both for their disease and for their lifestyle.”

Bronchitol was developed by Pharmaxis Ltd., and Chiesi is now its exclusive distributor in the United States and 11 countries in Europe. Bronchitol is also approved and marketed in Australia, Europe, Russia, and several other countries.

Growing in Cary

Chiesi is the American subsidiary of family-owned Chiesi Farmaceutici of Parma, Italy.

Globally, the parent company’s portfolio of nine marketed drugs is focused on acute cardiovascular care, neonatology, CF and other rare diseases. Chiesi is best known for Curosurf (poractant alfa), a lung surfactant for treating respiratory distress syndrome in premature infants.

In the U.S., it focuses on acute cardiology, neonatology and CF.

Its roots stateside go back to 2004, when Cornerstone BioPharma Holdings was formed in Cary to develop respiratory drugs. Through a reverse merger with Critical Therapeutics of Lexington, Mass., in 2008, Cornerstone became a publicly traded company and changed its name to Cornerstone Therapeutics.

Chiesi became a majority shareholder of Cornerstone in 2009. Five years later, it bought the remaining shares, taking Cornerstone private as its wholly owned U.S. subsidiary.

In 2019, it moved into its new headquarters, the top two floors of a new, six-story, office building — 55,000 square feet in all – overlooking the Koka Booth Amphitheater and Symphony Lake in Regency Woods II.

At the time, the company said it planned to double its 130-person Cary staff in the next 10 years.

Zwinski said it’s still on target.

“Over the next few years, we expect Chiesi USA will experience steady revenue and employee growth in both Cary and Boston,” he said. “This growth would be linked to strong performance expected from our growing portfolio of marketed products, launch of new specialty pipeline programs and support for our emerging rare-disease business unit.”

(C) N.C. Biotech Center