Reimbursement Agreement for New Cystic Fibrosis Drug Awaits EMA Approval

According to a story from the Irish Medical Times, Ireland’s Health Service Executive (HSE) has recently announced the extension of a reimbursement agreement between the agency and the pharmaceutical company Vertex in regards to its newly released triple-action therapy for cystic fibrosis called Trikafta. This latest therapy from the company combines three active components from its previously developed medicines that offer a potential breakthrough in quality of life for a significant portion of these rare patients.

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 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.

Waiting for Approval

Under terms of the agreement, all patients in Ireland that are aged 12 years or older can be reimbursed for treatment with Trikafta. However, this doesn’t mean that patients can get treated right away, because the drug still isn’t cleared for use in Ireland or the EU as a whole. Vertex submitted a Marketing Authorisation Application to the European Medicines Agency (EMA) in October of last year and is currently waiting on a verdict.

At this juncture, Trikafta is only approved for use to treat cystic fibrosis in the US for patients aged 12 or older that have the f508del mutation. In these patients, Trikafta has had a considerably greater effect than previously developed treatments, and may transform cystic fibrosis from a life-limiting disorder to a chronic condition that can be successfully managed. 


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