Top of page
Health

Cystic fibrosis Orkambi drug will ‘transform lives’ of young people

Colorful pills and capsules

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.

You might also like

Deaf patient use video conference, make online consultation by sign language with doctor on tablet Deaf patient use video conference, make online consultation by sign language with doctor on tablet

HHS and DOJ issue guidance on nondiscrimination in telehealth

The U.S. Department of Health and Human Services (HHS) and…

Woman silhouette with sun and clouds in her head Woman silhouette with sun and clouds in her head

Applications open for Diverse Communities Mental Health and Wellbeing Grants

Applications are now open for round two of the Diverse…

Close up of taking sample for coronavirus testing Close up of taking sample for coronavirus testing

Free RAT program for eligible Victorians with disability extended

The free rapid antigen tests program for eligible Victorians with…

The Yang-Tan Institute of Employment and Disability has joined a multi-institution team that has received a $5 million grant from the National Science Foundation (NSF) to help create better job outcomes for people with autism. The Yang-Tan Institute of Employment and Disability has joined a multi-institution team that has received a $5 million grant from the National Science Foundation (NSF) to help create better job outcomes for people with autism.

Research shows AI can improve stroke diagnostics

A new study presented on Thursday at the Society of…