FDA Approves Symdeko Tablets for Ages 6 and Older

JUNE 21, 2019
Laura Genn
FDA logoThe 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.

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