Cystic fibrosis drug Orkambi 'approved for use among 6-11 year olds'


The pharmaceutical firm making cystic fibrosis drug Orkambi says it has received EU approval to treat children.

Manufacturer Vertex says it will now be suitable for use for those aged 6 to 11.

It has already been licensed for patients 12 and over.

Earlier this year, the HSE agreed to make Orkambi available to Irish patients after major price negotiations with the pharma industry.

According to Vertex, Orkambi is the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of the F508del mutation.

In Europe, there are approximately 3,400 children ages six through 11 with two copies of this mutation.

“The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to ORKAMBI,” said Simon Bedson, senior vice president and international general manager at Vertex.

“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.”

Professor Marcus Mall, director of the Division of Paediatric Pulmonology & Allergology and the Cystic Fibrosis Centre at the Heidelberg University Hospital, Germany, said: “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.

"Studies of Orkambi in children ages 6 through 11 have shown improvements in clinically relevant outcomes, like lung function and weight gain.”

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