A ‘miracle’ cystic fibrosis drug was today approved for toddlers battling the cruel disease, sparking hope for hundreds of parents.
Until now, health chiefs had only approved Kaftrio for children over six.
But the Medicines and Healthcare products Regulatory Agency (MHRA) today gave the medication – thought to cost in the region of £200,000-a-year – the green light for youngsters two and above.
Studies have shown it can extend the life-expectancy of sufferers by 30 years.
NHS watchdogs are currently considering rejecting the drug for widespread NHS use due to its cost, however.
Nikki Thorbinson (right) was hoping her five-year-old boy Henry would get Kaftrio before Christmas
Henry, pictured, must attend countless appointments, take medications, have scans, X-rays and physiotherapy every day just to stay well
In draft guidance last week, National Institute for Health and Care Excellence (NICE) said Kaftrio shouldn’t be recommended despite its obvious benefits.
Only kids already on Kaftrio will be allowed to keep taking it if NICE doesn’t change its mind in the coming months.
Under the MHRA’s old two-tier licensing system, parents of toddlers battling cystic fibrosis feared they would never benefit from the drug.
Hundreds of families with children aged two to five, are now expected to come forward to get the drug before the supply is potentially cut-off.
Up to 600 children could benefit from the deal, it is estimated.
Ahead of today’s decision to licence Kaftrio for younger kids, affected families told MailOnline how they were terrified their youngsters may be denied the opportunity to get the drug because of their age.
Nikki Thorbinson, whose five-year-old son Henry has cystic fibrosis, was in ‘despair’ and ‘clinging to a glimmer of hope’ that it would eventually be made available.
She said: ‘This medication has been talked about by our teams for the last five years.
‘It’s given hope of a normal life freedom from the burden of the therapies that Henry endures a smile on his face each day.’
Giving Henry the drug would mean ‘he’s got a future to thrive’, she said.
Kaftrio is only currently available on the NHS through a temporary deal with London-based manufacturer Vertex Pharmaceuticals.
Pharmaceutical bosses agreed to lower their price for the agreement, which runs out in August 2024. The exact amount hasn’t been shared publicly.
Despite being given Kaftrio slightly cheaper than its listed price-tag, NICE last week announced the ‘triple-combination therapy’ and two others (Symkevi and Orkambi) were not ‘cost-effective’.
Draft NICE guidance revealed its cost-benefit ratio was above what was considered ‘an acceptable use of NHS resources’.
A final ruling is expected in March 2024, with committee members set to meet next month to discuss the evidence further.
John Stewart, national director for specialised commissioning at NHS England, said: ‘Children as young as two years old with cystic fibrosis will now be eligible to receive Kaftrio.
‘Cystic fibrosis centres across the country have plans in place to ensure all children eligible today can be provided long-term access to this life-changing treatment.’
If NICE officially rejects Kaftrio, it means anyone not already taking the drug will not be eligible to get it on the NHS.
It’s hoped the drug Kaftrio, pictured, which is a triple therapy — which combines three compounds, ivacaftor, tezacaftor and elexacaftor — will add decades to the lives of people with cystic fibrosis
The Cystic Fibrosis Trust hopes ‘everyone will move as quickly as possible to ensure eligible children can receive Kaftrio quickly’.
David Ramsden, the charity’s chief executive, said: ‘This is great news and another important step in ensuring access to life-changing modulator drugs for all of those with cystic fibrosis who could benefit.
‘I’m also reassured that NHS England has confirmed all children eligible today can be confident about their long-term access to these treatments.
‘Today’s news reinforces the need to ensure that the current NICE process rapidly results in a comprehensive deal to end the uncertainty for all who could benefit in the future.’
Cystic fibrosis — which affects 11,000 people in the UK — is usually detected at birth using a newborn screening heel prick test.
It is caused by a faulty gene which a child inherits from both parents, which makes it difficult for cells to move water and salt in and out of cells.
As a result, the body produces thick, sticky mucus that clogs the digestive system, creating problems with food absorption, and the lungs, which results in infections and scarring that gradually reduces patients’ ability to breathe.
This slow suffocation is often what ultimately kills.
Treatment advances over the past four decades have boosted life expectancy – as recently as the 1970s, few people with cystic fibrosis lived beyond their teens.
Kaftrio corrects the underlying problem by helping to create thinner mucus, thereby relieving symptoms.
Experts are hopeful the triple therapy — which combines ivacaftor, tezacaftor and elexacaftor — will add decades to the lives of people with cystic fibrosis.
WHAT IS CYSTIC FIBROSIS?
Cystic fibrosis is an incurable genetic disease that affects around 70,000 people worldwide and more than 10,900 people in the UK.
You are born with CF and cannot catch it later in life, but one in 25 of us carries the faulty gene that causes it, usually without knowing.
The defective gene causes a build-up of mucus in the airways, making it increasingly difficult to breathe over time.
Mucus also blocks the natural release of digestive enzymes, meaning the body does not break down food as it should.
Signs and symptoms:
- A persistent cough that produces thick mucus (sputum)
- Exercise intolerance
- Repeated lung infections
- Inflamed nasal passages or a stuffy nose
While healthy people cough naturally, that does not happen for people with CF.
Eventually, lung function depletes to the point that sufferers will need a double lung transplant to survive.
How is cystic fibrosis treated?
Medicines including CFTR modulators like Kaftrio, Symkevi, Orkambi and Kalydeco are used to treat CF.
Kaftrio is first triple combination therapy to treat the cause of CF. It is licensed for people with CF aged 6 years and older, with at least one F508del mutation.
Physiotherapy is also important for airway clearance to help loosen and remove the mucus that builds up in the lungs, or maintaining good posture to avoid back problems that can have a negative impact on lung function.
Exercise can also help clear mucus from the lungs, improve physical bulk and strength, and help improve overall health.
A person with CF needs to have a balanced diet, but may also need to calories to maintain a healthy body weight.
How does CF affect female fertility?
Women with CF are more likely to experience irregular periods or the absence of their period altogether, particularly if ill or very underweight.
This means they may not ovulate (produce an egg for fertilisation) each month.
Women with CF may also have thicker vaginal mucus, which can make it harder for the sperm to reach the egg.
Source: Cystic Fibrosis Foundation and Cystic Fibrosis Trust