New Cystic Fibrosis drugs could take months
The parents of two children who have Cystic Fibrosis say they're disappointed they won't have access to the life changing drug Orkambi for up to 6 months- despite the fact it's now available on the NHS.
The drug was approved in the UK in October after years of campaigning by patients. Parents Jodie and Shane were told their 4-year-old son Sebe was eligible for the drug and they understood he would be prescribed it by Christmas. However, they have now been told they could be waiting for up to six months.
Dad Shane remembers the moment he found out that Sebe and his daughter Ruby, who also has the condition, would finally have access to Orkambi. He described the moment as "the best day ever" other than the day his children were born.
The couple say that they feel "frustrated" and "angry" following the news that their children would not have immediate access to Orkambi, especially after four years of campaigning for it.
What is Cystic Fibrosis?
Cystic fibrosis is a genetic condition, which affects around 10,000 people in the UK.
The gene affected by CF controls the flow of salt and water in and out of the cells and causes a sticky mucus to build up in the lungs and digestive system.
About half of people with cystic fibrosis will live past the age of 40. Children born with the condition today are more likely to live longer than this.
What is Orkambi?
Orkambi is a combination medicine, made up of the two drugs 'ivacaftor' and 'lumacaftor'. It treats a mutation which is present in around 50% of people with Cystic Fibrosis in the UK.
The drug helps bring more proteins to the surface of cells in the body and open chloride channels to operate more effectively. This means the lungs have a healthier balance of salt and water.
Research shows that Orkambi can slow decline in lung function - the main cause of death among people with cystic fibrosis - by 42%.
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