Medication Monitor



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

Lumacaftor/ivacaftor

(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.