Unmet Medical Need
While there is no cure for the disease, thanks to medical advances, a majority of cystic fibrosis sufferers today reach adulthood. The goal for doctors treating CF sufferers is to hydrate, breakdown and move the excessive, sticky mucus secretions to improve lung function and reduce the number and severity of secondary lung infections. CF sufferers and their carers are generally able to manage the condition at home using a combination of exercise daily physiotherapy, postural drainage, and chest percussion (to assist the sufferer to expel mucus from their lungs). Depending on the severity of the condition, caring for a person with CF can take several hours of at-home treatment every day.
Medications to treat CF are limited, and while newer medications targeting the defective protein are now available they are not beneficial in all patients. Nebulised medications, delivered by aerosol or a face mask, are used to make the mucus less thick and sticky and open up the airways. Antibiotics, either nebulized or by oral or intravenous administration may also be required to treat secondary infections.
The treatment of cystic fibrosis by clinical teams is highly individualized. The need to consider the individuals’ current health/disease state, any comorbidities, as well as important lifestyle factors, makes treatment complex and varied. Having a broad range of therapies available for use in a highly tailored way helps to optimize outcomes for CF patients.