The FDA has expanded its approval of cystic fibrosis treatments

The U.S. Food and Drug Administration today expanded the adaptation of Symdeko, a tezacaftor/ivacaftor tablet, to treat pediatric patients 6 years of age and older with cystic fibrosis and certain genetic mutations. Last year, the U.S. Food and Drug Administration approved Symdeko's treatment of patients 12 years and older with the same specific gene mutation.“ Decades ago, patients with cystic fibrosis were usually expected to live to 10 years old, and few lived to ad her youth. Since then, while extensive research into the disease has led to more treatments for the debilitating disease, increasing life expectancy and improving the quality of life of patients, there has been no cure," said Banu Karimi-Shah, M.D., acting deputy director of the Lung, Allergy and Rheumatology Products Division at the FDA's Drug Evaluation and Research Center. Today, Symdeko's approval of children as young as 6 years of age provides an important treatment option for young patients and provides more context for the safety and dosage specific to this population. The U.S. Food and Drug Administration remains committed to advancing new treatment options in areas that do not meet the needs of patients, especially in diseases affecting children. cystic fibrosis is a serious genetic disorder that forms thick mucus in the lungs, digestive tract and other parts of the body. It can lead to severe respiratory and digestive problems as well as other complications such as infection and diabetes. Cystic fibrosis is caused by defective proteins caused by mutations in the cystic fibrosis transfilm conduction regulation factor (CFTR) gene. The CFTR gene has about 2,000 known mutations. Symdeko is used to treat patients with two of the most common mutation types -- the F508del mutation -- or at least one mutation in the CFTR gene, which responds to Symdeko's active ingredients and/or has a clinical basis based on in-body data. Patients with cystic fibrosis and their caregivers should communicate and test with healthcare professionals to see what genetic mutations the patient has and whether Symdeko may work for them.Symdeko's efficacy in patients with cystic fibrosis aged 12 years and older was assessed in three phase 3, double-blind, placebo-controlled trials that showed improved lung function and other key indicators of the disease, including a reduction in acute seizures (a) a worsening of symptoms requiring treatment. The efficacy of patients aged 6 to 12 was inferred from patients 12 years of age and older, and additional support was provided for data from patients aged 6 to 12 years.Symdeko's safety in treating patients with cystic fibrosis between the ages of 6 and 12 was supported by a study that included a 24-week open treatment period in which 70 patients aged 6 to 12 had cystic fibrosis and similar safety observations in clinical trials in patients 12 years and older. Symdeko should always be taken with foods containing fat and certain antibiotics and epilepsy medications should be banned. As shown on the label, maltose, or food containing grapefruit or Seville oranges. Symdeko's prescription information includes warnings about elevated enzymes (transaminases) in the livers of people taking Symdeko, warnings about cataract risk in children who use another liver enzyme called cytochrome P450 3A4 (CYP3A). Before starting Symdeko, patients and their caregivers should discuss these risks with healthcare professionals and any medications they take. The most common side effects include headache, nausea, nosebleeds and dizziness.The safety and effectiveness of Symdeko in patients under 6 years of age with cystic fibrosis has not been studied.The U.S. Food and Drug Administration approved the application for priority review. Symdeko of Vertex Pharmaceuticals was approved. (Compiled by this web)