According to a story from European Pharmaceutical Review, the European Commission has recently approved a label extension of ORKAMBI (lumacaftor/ivacaftor), a drug used to treat cystic fibrosis. Under the extension, the medicine is now approved for patients between age one and two years. This allows the drug to be used in younger patients than before, potentially delaying the onset of cystic fibrosis symptoms.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the buildup of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. More useful treatments for the disorder have been introduced in recent years. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
Improving Treatment Outcomes
The expanded label was previously cleared in the UK last March. The two-part combination drug works to both clear mucus from and hydrate the patient’s airways. The drug is designed to treat the disease in patients with dual copies of the F508del mutation of the CFTR gene. This is the most widespread mutation in this disease. The treatment boosts expression of the mature CFTR protein and improves the protein’s ability to transport water and salt through cell membranes.
Overall, this expansion is good news for cystic fibrosis patients living in the European Union, who will now be able to begin treatment with ORKAMBI earlier and experience superior treatment outcomes.
