Vertex’ CF therapy Orkambi cleared for younger patients

European regulators have expanded the scope of Vertex’ Orkambi to include children with cystic fibrosis aged six through 11 who have two copies of the F508del mutation.

Orkambi (lumacaftor/ivacaftor) was first cleared for use in Europe back in 2016 for patients aged 12 years and older.

The firm says the drug’s expansion is based on data from two Phase III studies showing statistically significant improvements in lung function among children treated with Orkambi compared to placebo.

Improvements in body mass index (BMI) and the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score were also observed, although they were not statistically significant, the firm noted.

The therapy’s safety profile was also deemed acceptable, the most common adverse events having found to be infective pulmonary exacerbation, productive cough, nasal congestion, oropharyngeal pain, abdominal pain upper, headache, upper respiratory tract infection and sputum increased.

"A principal goal of treating CF is slowing the progressive lung damage caused by this life-shortening genetic disease while improving health in the short term," said Professor Marcus Mall, director of the Division of Pediatric Pulmonology & Allergology and the Cystic Fibrosis Center at the Heidelberg University Hospital, Germany.

"Studies of Okambi in children ages six through 11 have shown improvements in clinically relevant outcomes, like lung function and weight gain."

In Europe there are approximately 3,400 children ages six-11 who have two copies of the F508del mutation.

Vertex says existing reimbursement agreements in countries like Ireland will enable rapid access to the therapy, and that country-by-country reimbursement processes will now begin in other countries.

Orkambi was rejected for NHS use by the National Institute for Health and Care Excellence in 2016, because of uncertainties over its impact on lung function, particularly in the long-term.