Fighting for the drug that pauses cystic fibrosis
Cystic Fibrosis is a life-reducing genetic disorder and while outcomes have improved, half of those with it do not live beyond 40.
Orkambi is a drug that targets a mutation that around 50% of people with cystic fibrosis in the UK have. It has been shown to improve lung health by up to 42%.
Licensed for use in the UK since 2015, it costs around £105,000 per patient per year so is not available on the NHS, except for certain people on compassionate grounds.