Lifesaving cystic fibrosis drug now approved for 6-11 year olds
The life-saving cystic fibrosis drug, Kaftrio, has been approved for use for many eligible children aged 6-11 in England, Wales, Scotland and Northern Ireland. This is a huge milestone in the Kaftrio journey following years of campaigning by people with cystic fibrosis and their families.
Cystic Fibrosis Trust are delighted at the positive impact this will have for many children and their families. Cystic fibrosis is a progressive disease, so this is an important next step in limiting lung damage and slowing long term decline in health.
Catherine Farrer, from London, is the mother of Kate, aged 7, who has cystic fibrosis. “We’ve waited 7 and half years for this moment, never losing hope that we would be able to access medication that will hopefully make Kate’s journey through life that much easier. As we sit in hospital having IVs for the third time in 13 months, it is impossible to put into words what this means for not just Kate, but for our whole family, extended family, friends, medical team and everyone who plays a part in trying to keep Kate well and living a “normal” life.”
This announcement also comes at a timely point in the year. With rising living costs, and worries about respiratory infections, this is a challenging winter for many families of children with cystic fibrosis and the announcement will bring much-needed hope at a difficult time.
Stacey, from Cornwall, is the mother of Katy, aged 8, who has cystic fibrosis and is currently on two nebulisers a day, which goes to as high as three or four when she is symptomatic. “Having access to Kaftrio changes Katy’s future. Although Katy has fared well in her life compared to others. We still feel the stresses when it comes to doing physiotherapy or having tests at the hospital. One of the hardest things for Katy is to gain weight. She wishes that one day she doesn’t have to take creon anymore. Katy suffers with needle phobias, so annual review and blood tests take months of preparation with the psychologist. This is exhausting for her and emotionally exhausting for us to have to watch her go through it.”
David Ramsden, Chief Executive at the Cystic Fibrosis Trust, said: “Today the MHRA and European Commission has agreed to license Kaftrio for use in children aged 6- 11 years old. This means that now over 1,500 children across the UK and Northern Ireland stand to benefit from this drug and limit the damage CF does in these critical early years.
While this is a good day, we know that this is not a cure, and that the treatment does not work for every child. The Cystic Fibrosis Trust won’t stop until everyone with CF has access to all of the support and treatments they need.”