Vertex's Symdeko gains expanded US approval for use in younger children with cystic fibrosis

The FDA announced Friday that it has broadened the label for Vertex Pharmaceutical's Symdeko to include treatment of paediatric patients with cystic fibrosis ages six years and older who carry certain genetic mutations. The therapy, which combines Vertex's Kalydeco (ivacaftor) with tezacaftor, gained US approval last year to treat the underlying cause of cystic fibrosis in patients aged 12 years and older with two copies of the F508del mutation in the CFTR gene or who have at least one mutation that is responsive to the drug combination. 

Banu Karimi-Shah, acting deputy director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA's Center for Drug Evaluation and Research, said the new approval "provides an important treatment option for younger patients, and also provides more context on the safety and dosing specific to this population." 

Symdeko's initial approval for use in patients aged 12 years and older was based on data from three Phase III trials that showed improvements in lung function and other key measures of the disease, including a reduction in exacerbations. In regards to the expanded indication, the agency said efficacy findings were extrapolated to patients ages six to 12 years, while additional support came from data that included a 24-week treatment period with 70 cystic fibrosis patients ages six to less than 12 that yielded "similar observations of safety" to clinical trials involving older participants. 

The FDA noted that the safety and efficacy of Symdeko in patients with cystic fibrosis younger than six years of age have not been studied. 

Symdeko is Vertex's third medicine approved to treat the underlying cause of cystic fibrosis, following FDA clearance in 2015 of Orkambi (lumacaftor/ivacaftor) and the approval of Kalydeco in 2012.  

To read more Top Story articles, click here.