FDA Expands Cystic Fibrosis Treatment for Use in Children Aged 2 to 5 Years


Lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals) is the first drug approved to treat the underlying cause of cystic fibrosis in children aged 2 to 5 years old.

Officials with the FDA have updated the label of lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), approving the treatment's use in children aged 2 to 5 years old; with this approval, it is now the first drug to treat the underlying cause of cystic fibrosis (CF) in this population, according to a press release.

Lumacaftor/ivacaftor is indicated for the treatment of CF in patients who have 2 copies of the F508del-CFTR mutation, the most common genetic form of the disease. The treatment has already been approved for use in patients aged 6 years and older.

This approval is based on data from a phase 3 trial involving 60 patients that showed treatment with lumacaftor/ivacaftor was safe and well tolerated for 24 weeks in this patient population. According to the data, patients demonstrated improvements in sweat chloride, a secondary endpoint, at week 24, as well as changes in key growth parameters.

Lumacaftor/ivacaftor demonstrated a safety profile similar to that in patients aged 6 years and older. The most common adverse effect associated with its use was cough and most adverse effects were mild or moderate in severity. In the trial, 4 patients experienced serious adverse events and 3 patients discontinued treatment due to treatment emergent adverse events or elevated liver function tests.

Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface, according to Vertex.

“Cystic fibrosis is a systemic, multi-organ, progressive disease that is present from birth,” study author Jon McNamara, MD, medical director of the cystic fibrosis program at Children’s Minnesota hospital, said in a statement. “Research suggests Orkambi could impact CF outcomes in patients as young as 2 years old. This approval is a significant development that enables physicians to begin treating the underlying cause of the disease in this population earlier than ever before.”

Lumacaftor/ivacaftor oral granules are available in 2 dosage strengths for weight-based dosing: lumacaftor 100 mg/ivacaftor 125 mg and lumacaftor 150 mg/ivacaftor 188 mg.


FDA Approves ORKAMBI® (lumacaftor/ivacaftor) as First Medicine to Treat the Underlying Cause of Cystic Fibrosis for Children Ages 2-5 Years with Most Common Form of the Disease [news release]. Vertex’s website. https://investors.vrtx.com/news-releases/news-release-details/fda-approves-orkambir-lumacaftorivacaftor-first-medicine-treat. Accessed August 7, 2018.

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