The family of a Bury baby with cystic fibrosis are appealing for access for her to have lifesaving drug treatment deemed "too expensive" to have it on the NHS.

Eleven-month-old Isabelle Bennett was diagnosed with the genetic condition just three weeks after she was born at St Mary's Hospital in Manchester. 

Isabelle has ongoing treatment at Royal Manchester Children's Hospital including daily physiotherapy to manage the condition as well as medication to treat symptoms affecting the absorption of fat by the pancreas. 

Cystic fibrosis is a genetic disease which causes mucus to build up in the lungs and digestive systems.

Symptoms worsen over time and Isabelle’s parents Hannah and Matthew fear her life will be severely shortened without lifesaving medication.

The little girl was due to start having a modulator drug, Orkambi, just before Christmas, but her parents have now been told the drug is "too expensive" on the NHS. 

Isabelle would ideally take Orkambi until she is old enough for another drug, Kaftrio.

Hannah, 31, said she had hoped that another drug, Kaftrio, would be approved for use for two-year-olds before being told it could be deemed too expensive for new patients, despite its potential to extend Isabelle's life significantly.

READ MORE:  Ainsworth Isolation Hospital volunteer speaks on her time there

Bury Times: Isabelle Bennett was diagnosed with cystic fibrosis at three weeks oldIsabelle Bennett was diagnosed with cystic fibrosis at three weeks old (Image: Hannah Bennett)

Hannah says Isabelle must be put on medication as soon as possible or her condition “will kill her as a teenager".

"At the moment, she's not got an understanding and we carry the burden of her illness," Hannah said. 

"We're an open family, I've also got a four-year-old, Harry, and we talk to him to get him to understand." 

READ MORE: Bury LGBTQI+ group given Kings Award for Voluntary Service

Draft guidance published by NICE says that while clinical evidence shows the drugs "improve lung function, growth, weight gain and reduces the number of lung infections" it has concluded that cost estimates "are above the range that NICE considers an acceptable use of NHS resources".

Hannah and Matthew are now appealing for anyone affected by cystic fibrosis to submit comments to NICE’s online consultation in the hope of making the drugs available for use.

They have also urged people to sign a petition to the government to campaign for access to these lifesaving drugs. 

Hannah added: “Our only hope is to shout from the roof tops to help children like my daughter get the lifesaving medication they deserve.

"I want my daughter to have a chance to allow her to be a child and grow up planning her future.

"To my daughter I am her world, but to the world I am a nobody. I am just a mother willing to do whatever it takes to save the life of my child. I will not give up.

“These drugs must be made available, the earlier we get this medication the better."

Helen Knight, director of medicines evaluation at NICE, said: "We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure that taxpayers continue to get value for money after interim access where further data was collected.

"The committee want to hear from stakeholders through consultation on important aspects of its draft conclusions.

"This is so that we have all the relevant information to accurately capture the value of these effective medicines when the committee makes its final decision.

"We are continuing to work collaboratively with the company, NHS England and other stakeholders including the Cystic Fibrosis Trust to deliver the best outcome both for people with cystic fibrosis and for the wider NHS.

"Existing patients and new patients who are started on treatment while the NICE evaluation is ongoing will continue to have access to the treatments after NICE has issued its final recommendations irrespective of the outcome.”