On Monday, the FDA (Food and Drug Administration) has legalized a newly discovered medication for people suffering from cystic fibrosis. The therapy, available for patients above the age of 12, may benefit around 90% of patients having cystic fibrosis. The newly FDA-approved Trikafta is a combination of three therapies – ivacaftor, elexacaftor, tezacaftor. It is the first time; the FDA has permitted the use of a medication for such a major group of patients. The effort also marks the substantial step medicine has made in CF treatment over the past hundred years. Besides, the therapy is available with a minimum of one F508del mutation. It is the most popular mutation in cystic fibrosis patients.
The trio elements present in the combination remedy function together to assist in enhancing operation in the CFTR protein. This protein is found damaged in most of the cases of cystic fibrosis. Sometimes, it does not work as expected to function. Due to this CFTR protein deficiency, patients suffer from an increased amount of mucus in their lungs. Besides, the material is unusually dense and sticky; that can result in complexity to breathe, followed by infection. In some cases, the complexity may result in death.
Since its emergence, Trikafta has two large-scale clinical trials have revealed its benefits. First among the two included 403 candidates, while the second involved 107 volunteers. Patients who participated in both trials had revealed different mutations, leading to separate CFTR functionality. Notably, the outcomes of both studies have shown improvements in the functioning of the lungs. Even more, the medication has proved effective in body mass index (BMI), sweat chloride, and the hallmark feature of CF.
Apart from the positive results, some participants have also experienced some side effects of the medication. The list of negative impacts included abdominal pains, headaches, diarrhea, upper respiratory tract infections, and rashes. The symptoms of more severe side effects included threatened liver function along with the risk of cataracts. Reportedly, Vertex Pharmaceuticals, manufacturer of Trikafta, has assured deployment of the medicine within two weeks.