FDA expands Vertex’s cystic fibrosis drug Symdeko in paediatric patients
pharmafile | June 24, 2019 | News story | Sales and Marketing | FDA, Symdeko, Vertex, cystic fibrosis, pharma
Vertex has announced that the FDA has chosen to expand the existing authorisation on its cystic fibrosis (CF) therapy Symdeko (tezacaftor/ivacaftor).
The drug received marketing approval in February last year in patients over 12 years old who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, or have at least one mutation that is responsive to tezacaftor/ivacaftor; this new approval extends the label to include patients as young as six years old.
The decision to expand the indication was based on data derived from three Phase 3 studies in patients aged 12 years and older, from which the drug’s efficacy in patients between the ages of six and 12 was extrapolated. The drug demonstrated “improvements in lung function and other key measures of the disease, including a reduction in exacerbations (a worsening of symptoms requiring treatment)”.
“Decades ago, patients with cystic fibrosis were generally expected to live until 10 years of age, with few surviving into their teenage years. Since then, wide-ranging research on the disease resulted in more treatments for this debilitating disease that have extended life expectancy and improved quality of life for patients, but there is still no cure,” explained Dr 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. “Based on their individual genetic makeup, individuals may respond differently to certain drugs, so it is important to provide a variety of options. Today’s approval of Symdeko for children as young as six 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.”
Matt Fellows
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