(Reuters) - Corbus Pharmaceuticals Holdings Inc said on Monday the U.S. Food and Drug Administration will evaluate its experimental cystic fibrosis drug without requiring proof the product improves lung function, dramatically cutting the time and cost it will take Corbus to develop the drug.
Companies developing drugs for cystic fibrosis, a life-threatening genetic disorder that damages the lungs and affects about 30,000 Americans, have historically been required to show an improvement in lung function based on a measure known as forced expiratory volume in one second, or FEV1.
The FDA will instead evaluate Corbus’s drug, Lenabasum, based on its ability to reduce disease flare-ups.
“It’s very significant that we can do something significantly smaller and shorter,” Yuval Cohen, the company’s chief executive officer, said in an interview.
Almost all cystic fibrosis patients are on treatment these days, he said, meaning a big trial would be needed to show an improvement over a patient’s baseline.
To show lung function improvement Cohen estimated the company would have had to enroll more than 1,500 patients and run the trial for a year. As it is, the company plans to test 415 patients for six months.
The FDA’s move is consistent with its commitment under President Donald Trump to cut drug development times and use new types of clinical trials to speed new drugs to the market.
The FDA has never allowed a reduction in disease flare-up by itself to be the main goal of a clinical trial for a cystic fibrosis drug.
Pulmonary exacerbations are a primary driver of disease worsening and death, Cohen said. The company is also testing the drug in systemic sclerosis, a connective tissue disease and dermatomyositis, an inflammatory condition that affects muscles. Lenabasum is designed to reduce inflammation without suppressing the immune system as some anti-inflammatories do.
Laura Chico, an analyst at Raymond James, said she expects Lenabasum to generate peak annual sales of nearly $2 billion if the company wins approval in all three conditions, with the earliest potential U.S. market entry in 2021.
Reporting by Toni Clarke in Washington; Editing by James Dalgleish