According to a story from england.nhs.uk, a recent agreement struck between the UK’s NHS and Vertex Pharmaceuticals will herald a major improvement for the many of the country’s cystic fibrosis patients. Because of this agreement, Vertex’s triple action combination therapy Kaftrio (known as Trikafta in the US) will now be covered under the NHS. The drug is effective for as much as 90 percent of patients and has been hailed as a significant treatment breakthrough.
About Cystic Fibrosis (CF)
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. 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 40’s and 50’s with good care. To learn more about cystic fibrosis, click here.
Making History
There are an estimated 7,000 people living with cystic fibrosis in the UK. The deal went forward as soon as the drug was officially cleared for use by European regulators. These patients will be among the very first in Europe to have access to the therapy.
About Kaftrio
Kaftrio has been found to be more effective than other medications developed for the disorder, with the UK Cystic Fibrosis Medical Association claiming that the drug could possibly be “truly life-transforming” for many patients. It may have the potential to change the disease from a life-shortening, progressive illness to a more manageable chronic condition.
The only downside is that Kaftrio is designed to work on the most common mutations associated with the disorder and around ten percent of patients have rarer mutations that are not impacted by the drug. These patients are still in dire need of more effective therapies.
Nevertheless, the UK cystic fibrosis community has a lot to celebrate with this historic announcement.
