Overview
Cystic fibrosis is an inherited disorder that primarily affects the lungs, but also affects the digestive system and other organs. Individuals with cystic fibrosis have a build-up of mucus in the lungs that leads to symptoms such as recurrent cough, lung infections, shortness of breath, and chronic difficulty breathing. Cystic fibrosis is progressive and can eventually lead to respiratory failure.
Symptoms
Rapid worsening of symptoms in individuals with cystic fibrosis (CF) are called pulmonary exacerbations (PEx).1
Symptoms of PEx may include:
- increased cough
- increase in mucus (called sputum)
- change in the color or consistency of sputum
- shortness of breath/exercise intolerance
- fatigue
- hemoptysis
Individuals with CF who have more episodes of PEx have been shown to have worse long-term health outcomes, such as increased risk of lung function decline, lung transplant, or death.2 Currently, there are no FDA-approved therapies for CF-related pulmonary exacerbations.
Aceragen is developing ACG-701, a novel oral antibiotic in the United States. ACG-701 is an investigational therapy for the acute treatment of cystic fibrosis-related pulmonary exacerbations (PEx).
We are conducting a Phase 2 clinical trial (the REPRIEVE study) to evaluate the safety and efficacy of ACG-701 in individuals with acute cystic fibrosis-related pulmonary exacerbations.
For more information on the REPRIEVE study, please refer to ClinicalTrials.gov.
Resources
Please note, these links are provided for informational purposes for patients, families, and healthcare providers. Aceragen does not endorse any particular organization or the content on their websites.
References:
- Goss, C. H. & Burns, J. L. Exacerbations in cystic fibrosis. 1: Epidemiology and pathogenesis. Thorax 62, 360-367, doi:10.1136/thx.2006.060889 (2007).
- de Boer, K. et al. Exacerbation frequency and clinical outcomes in adult patients with cystic fibrosis. Thorax 66, 680-685, doi:10.1136/thx.2011.161117 (2011).
