FDA Approves Symdeko Tablets for Ages 6 and Older

The US Food and Drug Administration (FDA) has expanded Symdeko tablets from its previous approval for ages 12 and older to include patients aged 6 years and older who have the same particular genetic mutations.

“Based on their individual genetic makeup, individuals may respond differently to certain drugs, so it is important to provide a variety of options,” said 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, in a statement.

Symdeko tablets, a combination of tezacaftor and ivacaftor, are used treat cystic fibrosis, a disease that was once known to shorten life expectancy to 10 years but can now be treated, although not cured.

“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,” said Karimi-Shah.

Data from a 24-week, open-label treatment trial period, during which 70 cystic fibrosis patients aged 6-12 years old received Symdeko tablets, supported the FDA’s decision to approve expansion to patients 6 years or older. The FDA also granted priority review to Vertex Pharmaceuticals Incorporated’s application for Symdeko.

Presently, no one has conducted research on the safety and/or efficacy of Symdeko in patients with cystic fibrosis who are younger than 6 years old.