Some 3,400 European children whose cystic fibrosis (CF) is caused by the CFTR F508del mutation may now receive Orkambi (lumacaftor/ivacaftor), thanks to the European Commission’s decision to extend the therapy’s marketing authorization.
The Jan. 10 extension, which applies to children aged 6 to 11, follows a positive recommendation issued in November 2017 by the Committee for Medicinal Products for Human Use (CHMP).
Vertex Pharmaceuticals, the Boston-based company that produces Orkambi, said it will initiate the country-by-country reimbursement process throughout the 28-member European Union, though children in Ireland and other markets with reimbursement agreements in place will be able to access Orkambi immediately.
“The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to Orkambi,” Simon Bedson, senior vice president and international general manager at Vertex, said in a press release. “Where these agreements are not in place, Vertex is committed to working with local authorities so those who could benefit from this medicine are able to do so as quickly as possible.”
The European Commission based its decision on safety and efficacy data collected from two Phase 3 studies (NCT02514473 and NCT01897233) of Orkambi in children with CF ages 6 through 11, who had two copies of the F508del mutation.
Orkambi significantly improved lung function, with better absolute change in lung clearance index and predicted forced expiratory volume in one second (ppFEV1) than placebo. In addition, treated patients gained weight and improved their Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score, although these were not statistically significant.
Common adverse events linked to Orkambi use among these children were infective pulmonary exacerbation, productive cough, nasal congestion, throat pain, abdominal pain, headache, upper respiratory tract infection and sputum increase.
“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 Dr. Marcus Mall, director of the Cystic Fibrosis Center at Germany’s Heidelberg University Hospital. “Studies of Orkambi in children ages 6 through 11 have shown improvements in clinically relevant outcomes, like lung function and weight gain.”
The U.S. Food and Drug Administration approved Orkambi in September 2016 to treat children ages 6 to 11 with cystic fibrosis.