Cystic fibrosis is a genetic disease of the respiratory tract that presents itself approximately in 1000 new cases each year. This consist in a mutation of a gene that changes the protein that regulates the movement of salt through the cells. This results in a buildup of mucus in the lungs and other organs as well, such as the pancreas. This mucus clogs the airways, causing several infections (because of bacteria getting trapped in the mucus), lung damage and death because of respiratory failure. Some symptoms of this disease are coughing with phlegm, frequent lung infections, difficulty breathing, wheezing, and bulky stools.
Up to date, there is no cure for cystic fibrosis, but there have been improvements regarding the care of quality of life of the patients. The average survival age is about 40 years. Patients are given care of regarding the clearance of their airways to help them get rid of the mucus that builds up in the lungs. Patients may use aerosols or vibrating vests that help in the thinning of the mucus. Some enzyme supplements also help improve the digestive symptoms also. There is currently a drug that has been approved by the FDA that is called ivacaftor. This drug improves the lung function. There is also another drug available, called Orkambi®, that combines ivacaftor with lumacaftor. These medications improve how the lungs are working.
There is currently a clinical trial from Vertex Pharmaceuticals to evaluate the effectiveness of this new drug VX-445 in combination with tezacaftor and ivacaftor. The trial is called AURORA program and will enroll 360 patients from 12 years and older. The pharmaceutical company is expecting that the information gathered from this trial will help support the submission of an NDA in the USA and Europe. At Althian, we are actively seeking and participating in trials where patients can have more options for their diseases that allow them to have a better quality of life.