FDA OKs Tezacaftor/Ivacaftor for Patients Ages 6 and Older with Cystic Fibrosis
This approval expands tezacaftor/ivacaftorâ€™s indication to include patients ages 6 and older with cystic fibrosis who have certain genetic mutations.
Officials with the FDA today expanded the indication for tezacaftor/ivacaftor (Symdeko, Vertex) tablets for the treatment of pediatric patients ages 6 years and older with cystic fibrosis (CF) who have certain genetic mutations, according to a press release.
Tezacaftor/ivacaftor was initially approved by the FDA in February 2018 for the treatment of CF in patients ages 12 years and older who had the same specific genetic mutations.
CF, a rare genetic disorder, is caused by a defective or missing protein that results from mutations in the CF transmembrane conductance regulator (CFTR) gene. The disease results in the formation of thick mucus that can build up in the lungs, digestive tract, and other parts of the body.
Tezacaftor/ivacaftor is designed to treat patients who have 2 copies of the F508del mutation, the most common type of mutation, or who have at least 1 of the mutations in the CFTR gene that is responsive to the active ingredients.
This approval is based on clinical data from 3 phase 3 studies that evaluated the efficacy of tezacaftor/ivacaftor in patients with CF ages 12 years and older and the safety in patients ages 6 to less than 12 years. The efficacy for patients ages 6 to 12 was extrapolated from patients ages 12 years and older, with additional support from data in patients ages 6 to 12 years.
According to the efficacy data, tezacaftor/ivacaftor demonstrated improvements in lung function and other key measures of disease, such as a reduction in exacerbations.
Based on the safety data, the safety profile of tezacaftor/ivacaftor was supported by a study that included 70 patients with CF ages 6 to less than 12, with similar observation in clinical trials of patients ages 12 and older.
The safety and efficacy of tezacaftor/ivacaftor have not been evaluated in patients younger than 6 years of age, according to the FDA.
“Based on their individual genetic makeup, individuals may respond differently to certain drugs, so it is important to provide a variety of options,” Banu Karimi-Shah, MD, acting deputy director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Today’s approval of Symdeko for children as young as 6 years old provides an important treatment option for younger patients, and also provides more context on the safety and dosing specific to this population. The FDA remains committed to advancing novel treatment options for areas of unmet patient need, particularly in diseases impacting children.”
Tezacaftor/ivacaftor should be taken with food that contains fat and never in combination with certain antibiotics, seizure medicines, St. John’s wort, or food containing grapefruit or Seville oranges, according to the label.
FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older [news release]. FDA. https://www.fda.gov/news-events/press-announcements/fda-expands-approval-treatment-cystic-fibrosis-include-patients-ages-6-and-older. Accessed June 21, 2019.