Posted from: Monday, July 15, 2019 - 04:01 PM - Present

Expanded Pediatric Indication for Symdeko

June 21, 2019 – The U.S. FDA has approved an expanded indication for Symdeko® (tezacaftor/ivacaftor and ivacaftor), manufactured by Vertex Pharmaceuticals. The combination therapy is now approved to treat cystic fibrosis (CF) in patients who are at least six years of age. Patients must be either homozygous for an F508del mutation, or have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that responds to tezacaftor/ivacaftor.

In addition to the expanded indication, the U.S. FDA approved a new, lower dosage strength for patients who weigh 30kg (66 pounds) or less. Symdeko originally came in packages that contained tezacaftor 100mg/ivacaftor 150mg combination tablets and ivacaftor 150mg tablets. It will now also be available in packages that contain tezacaftor 50mg/ivacaftor 75mg combination tablets with ivacaftor 75mg tablets.

Cystic fibrosis is a serious genetic disorder that leads to severe respiratory and digestive problems, as well as other complications such as infections and diabetes. According to the FDA, there are more than 2,000 known mutations of the CFTR gene, including the F508del mutation. In vitro data and/or clinical evidence are required to determine if Symdeko is appropriate for an individual patient based on which of these mutations are present in their DNA.

When first approved in 2018, Symdeko was indicated only for patients twelve years of age and up. Its efficacy in patients ages 6-12 years old was extrapolated from patients 12 years of age and up, with additional support from data in patients aged 6-12 years.

Recommended daily dosing is based on weight. One tezacaftor/ivacaftor combination tablet should be taken each morning, and one ivacaftor tablet should be taken each evening, approximately 12 hours later. Each dose should be taken with food that contains fat. Dosage adjustment may be required based on individual patient factors, such as hepatic health and any other medications the patient may be taking.

Last Updated Wednesday, October 23, 2019 - 07:35 PM.