According to a story from BioPortfolio, the biotechnology company Vertex Pharmaceuticals recently announced that the European Commission has approved a label extension for the company’s cystic fibrosis drug Orkambi. Under this label extension, the drug will now be approved to treat patients between the ages of two and five years that have the most common mutation associated with the disease. Vertex is committed to the development of therapies for serious, life-threatening diseases.
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up 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. 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.
This approval will allow a greater number of cystic fibrosis patients to be treated with Orkambi. It is estimated that there are around 1,500 kids in the EU who will be able to receive this drug for the first time. In addition, the ability for patients to be treated with the drug at an earlier age means that these patients could experience improved outcomes later in life.
The expansion of the drug’s label comes as the result of a Phase 3 open label study of 60 patients. In this study, treatment with Orkambi was well tolerated during the 24 week treatment period. The safety profile of the drug also did not differ significantly from earlier studies of patients aged six years or more.
This news should be regarded as another step forward for the cystic fibrosis community in Europe. Hopefully, it will lead to significant improvements in overall quality of life and lifespan for patients with this rare disorder.
