The lives of young people with cystic fibrosis will be “transformed” by a life-extending drug approved to treat the condition, campaigners have said.
Earlier this month , the Department of Health confirmed its intention to make Orkambi available in Northern Ireland. It improves lung function in those with cystic fibrosis – a life-shortening genetic condition that can cause fatal lung damage.
Approximately one in 2,500 babies born in the UK has cystic fibrosis. Only about half of those with the condition live to the age of 32.
Orkambi does not work for all patients, but two other drugs to treat cystic fibrosis symptoms, Symkevi and Kalydeco, will also be made available in Northern Ireland.
Symkevi is restricted to over 12-year-olds, while Kalydeco can be used from 12 months. Families have campaigned for Orkambi since 2015, when it was licensed to treat certain cystic fibrosis patients.
The drug can be given to children as young as two.The drug, which costs approximately £104,000 a year per patient, was deemed too expensive by the health advisory body, NICE.
Cystic fibrosis is an inherited condition in which the lungs and digestive system can become clogged with thick, sticky mucus. It can cause problems with breathing and digestion from a young age.