Medication Monitor

Generic Name (Trade Name—Company)
August 20, 2018


(Orkambi—Vertex Pharmaceuticals)
First medication approved to treat underlying cause of CF in children aged 2–5 years

Vertex Pharmaceuticals announced FDA approval of lumacaftor/ivacaftor to include use in children aged 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first medication approved to treat the underlying cause of CF in this population. 

The oral granules are available in two dosage strengths—lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg—for weight-based dosing.

FDA approval for this indication was based on a Phase III open-label safety study in 60 patients that showed treatment with lumacaftor/ivacaftor was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients aged 6 years and older. Improvements in sweat chloride, a secondary endpoint, were observed at week 24. Researchers also saw changes in key growth parameters, which were also secondary endpoints in the study.

The most common adverse event (≥30%) was cough (63%); most adverse events were mild or moderate in severity. Four patients experienced serious adverse events (two pulmonary exacerbations, one gastroenteritis, one constipation), and three patients discontinued treatment due to treatment-emergent adverse events or elevated liver function tests. 

The agent was originally approved for treatment of CF in patients aged 6 years and older who have two copies of the F508del-CFTR mutation.