Cystic Fibrosis

Cystic fibrosis (CF) is a chronic, rare, genetic disease caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF affects approximately 70,000 people in the U.S. and Europe.1

In people with CF, thick secretions build up in the lungs, pancreas and other organs. In the lungs, the mucus blocks airways, making it easy for bacteria to grow and infections to occur. These infections can severely damage the lungs over time and lead to respiratory failure.2 People affected by CF may have trouble digesting their food and may develop diabetes as a complication due to the disease's effect on the pancreas.3

A person with CF may also experience pulmonary exacerbations (PEx), which are an acute worsening of inflammation in the lungs with an increase in respiratory symptoms (for example, cough, shortness of breath) accompanied by an acute decrease in lung function.4 PEx are responsible for about half of long-term decline in lung function experienced by people with CF. More exacerbations are associated with greater lung function decline.5 Nearly 1 in 3 people with CF require treatment for PEx in any given year, and treatment success of PEx is currently described as "suboptimal."6,7 PEx can cost up to $120K per year in people with severe lung disease and are associated with higher one-year risk of death.8,9

Despite the major advances in treatment of CF over the last several decades, there has been a minimal reduction in the proportion of individuals who have PEx treated with IV antibiotics.6 Several classes of drugs have been considered to treat the underlying inflammation, though ibuprofen is the only anti-inflammatory drug currently recommended for the long-term treatment of CF airway inflammation. Despite this recommendation, very few eligible patients are prescribed ibuprofen because of side effects and monitoring requirements.6,10

James F. Chmiel, M.D., MPH Explains Cystic Fibrosis and the Effects of Inflammation and Fibrosis

Corbus Pharmaceuticals Holdings, Inc.
Corbus Pharmaceuticals Holdings, Inc.
Corbus Pharmaceuticals Holdings, Inc.

1. Health Advances, LLC Analysis
2. “About Cystic Fibrosis.” CF Foundation, Cystic Fibrosis Foundation, www.cff.org/What-is-CF/About-Cystic-Fibrosis/
3. “Cystic Fibrosis-Related Diabetes.” CF Foundation, Cystic Fibrosis Foundation, https://www.cff.org/Life-With-CF/Daily-Life/Cystic-Fibrosis-Related-Diabetes/
4. “Pulmonary Exacerbations Clinical Care Guidelines.” CF Foundation, Cystic Fibrosis Foundation, https://www.cff.org/Care/Clinical-Care-Guidelines/Respiratory-Clinical-Care-Guidelines/Pulmonary-Exacerbations-Clinical-Care-Guidelines/
5. Waters, Valerie, et al. “Effect of Pulmonary Exacerbations on Long-Term Lung Function Decline in Cystic Fibrosis.” European Respiratory Journal, vol. 40, no. 1, Jan. 2011, pp. 61–66., doi:10.1183/09031936.00159111
6. 2016 Patient Registry Annual Data Report. Cystic Fibrosis Foundation, 2016 Patient Registry Annual Data Report, www.cff.org/Research/Researcher-Resources/Patient-Registry/2016-Patient-Registry-Annual-Data-Report.pdf
7. Schechter, Michael S. “Reevaluating Approaches to Cystic Fibrosis Pulmonary Exacerbations.” Pediatric Pulmonology, 6 July 2018, doi:10.1002/ppul.24125
8. Rubin, Jaime L., et al. “Frequency and Costs of Pulmonary Exacerbations in Patients with Cystic Fibrosis in the United States.” Current Medical Research and Opinion, vol. 33, no. 4, 9 Feb. 2017, pp. 667–674., doi:10.1080/03007995.2016.1277196
9. Aaron, Shawn D., et al. “A Statistical Model to Predict One-Year Risk of Death in Patients with Cystic Fibrosis.” Journal of Clinical Epidemiology, vol. 68, no. 11, Nov.2015, pp. 1336–1345., doi:10.1016/j.jclinepi.2014.12.010
10. Cantin, André M., et al. “Inflammation in Cystic Fibrosis Lung Disease: Pathogenesis and Therapy.” Journal of Cystic Fibrosis, vol. 14, no. 4, July 2015, pp. 419–430., doi:10.1016/j.jcf.2015.03.003