Cystic fibrosis drug approved for younger patients

October 3, 2016

FDA recently expanded the use of lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals) to treat cystic fibrosis in children aged 6 to 11 years who have two copies of the F508del mutation.

FDA recently expanded the use of lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals) to treat cystic fibrosis in children aged 6 to 11 years who have two copies of the F508del mutation.

People with this mutation represent the largest population of those with CF, a rare, life-threatening disease. Orkambi is the first and only medicine to treat the underlying cause of CF for people with this mutation, according to Vertex.

Related: New cystic fibrosis drugs may significantly increase pharmacy benefit costs

Orkambi was previously approved by FDA for use in people aged 12 years and older with two copies of the F508delmutation. With this approval, approximately 11,000 people with CF in the United States are eligible for treatment with Orkambi.

Vertex also plans to submit a Marketing Authorization Application (MAA) variation in the European Union in the first half of 2017 for children aged 6 to 11 years who have two copies of the F508del mutation.

"The ability to treat children as young as six who have the most common form of the disease is an important milestone as we pursue our goal to develop medicines for all people with CF," said Jeffrey Chodakewitz, MD, executive vice president and chief medical officer at Vertex. "We believe it is important to treat the underlying cause of the disease as early as possible in these patients."

Related: FDA approves Orkambi for treatment of cystic fibrosis

The approval is based on data from a previously announced open-label phase 3 clinical safety study of Orkambi presented at the 39th European Cystic Fibrosis Society Conference in June.

While Vertex has had success with Orkambi, it lowered its revenue projections for the drug for 2016 from between $1 and $1.1 billion to between $950 million and $990 million. 

Serious adverse reactions to Orkambi include high liver enzymes in the blood, which can be a sign of liver injury, respiratory events such as shortness of breath or chest tightness, an increase in blood pressure and cataracts in some children and adolescents receiving Orkambi and ivacaftor, a component of Okrambi.

The most common side effects of Orkambi include shortness of breath and/or chest tightness; upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose; gastrointestinal symptoms including nausea, diarrhea, or gas; rash; fatigue; flu or flu-like symptoms; increase in muscle enzyme levels and irregular, missed, or abnormal menstrual periods and heavier bleeding.

Read more: FDA approves Orkambi for treatment of cystic fibrosis