Cystic fibrosis: Essex family unsure if son can use drug

The mother of a four-year-old boy with cystic fibrosis fears her son will never have access to the so-called “miracle drug” that could prolong his life.

Doctors do not expect Jesse, from Ingatestone, Essex, to live into his 30s.

But for the family, uncertainty remains as they don’t know if Jesse will qualify.

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the drug for children aged two to six, but it still needs the go-ahead from the National Institute for Healthcare Excellence (NICE).

Jessie’s mother Chloe told BBC Essex: “In the cystic fibrosis community we call this a life-saving drug, a miracle drug – the drug everyone wants their child to take. Masu.

“If Jesse takes this medication, he will probably need less physical therapy and will be able to get his life back.

“He doesn’t have a lot of energy at the moment. He can’t do as much as the other kids.”

Kaftrio was already approved to treat children 6 years and older with some types of cystic fibrosis.

This medication is given as a sachet of granules to be mixed with soft food.

Jesse’s strict routine involves waking his family up at 6:30 a.m. so he can take seven medications, do physical therapy, and do breathing exercises.

“One pill could change his life overnight,” Chloe said.

“It’s really hard because you look at the six-year-olds who are participating in this and you see their lives changing.

“When I hear that cough in the middle of the night, I know the clock is ticking and Jesse’s lungs are getting worse as time goes on.

“We will not stop fighting to get our son this medicine.”

A Department of Health and Social Care spokesperson said the department was investing more than £1.1 billion into research into rare diseases.

He added: “It is vital that patients have access to innovative new medicines like this, but the NHS must use its budget fairly and to the benefit of all patients.”