Wider Approval for Cystic Fibrosis 'Miracle' Drug

Dr Sheena Meredith

January 12, 2022

The Medicines and Healthcare products Regulatory Agency (MHRA) this week extended the licence for Kaftrio (Vertex Pharmaceuticals), a disease-modulating therapy for cystic fibrosis (CF), to children under 12. 

Previously licensed for older children and adults with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the drug is the latest in a suite of cystic fibrosis trans-regulatory (CFTR) modulator therapies that have transformed CF treatment and led to significant improvements in both lung function and quality of life, with the potential for a real impact on life expectancy.

Kaftrio is now approved on the same terms for children aged 6 to 11, meaning more than 1300 children in England are newly eligible to receive the drug, according to the NHS.

A spokesperson for the Cystic Fibrosis Trust, which has long campaigned for improved access to CFTR modulator drugs, described the development as "very exciting".

Life-Changing Drug, According to NHS Chief

According to the NHS, 9 in 10 patients with CF – representing more than 7000 people in England – can benefit from the 'triple therapy', which tackles the underlying causes of the disease as well as symptoms. 

NHS chief executive Amanda Pritchard described Kaftrio as an "incredible treatment".

"Since NHS staff delivered one of the fastest rollouts of Kaftrio in the world just over a year ago, the lives of thousands of patients with cystic fibrosis have been transformed," she said. "Innovative treatments like Kaftrio are life-changing for patients and their families."

Kaftrio has three active ingredients, elexacaftor (100 mg) and tezacaftor (50 mg), which increase the number of CFTR proteins on the cell surface, and ivacaftor (75 mg), which improves the activity of the defective CFTR protein. It is indicated in a combination regimen with ivacaftor alone, with a dosing regimen of 2 tablets of Kaftrio in the morning, taken with a fat containing food, and one ivacaftor tablet (150 mg) in the evening, approximately 12 hours later.

A spokesperson for Vertex Pharmaceuticals (Europe) told Medscape UK that the label extension submissions were supported by an open-label global phase 3 study in 66 children aged 6 through 11 years. Treatment with Kaftrio plus ivacaftor led to a mean 10.2% increase in percent predicted forced expiratory volume in one second (ppFEV1) at week 24, with improvements seen as early as 2 weeks after initiation. 

Improvements were also seen in CF respiratory questionnaire scores, lung clearance index, and sweat chloride concentration, while BMI, BMI-for-age z-score, weight, weight-for-age z-score, and height also increased over the treatment period, with height-for-age z-score maintained.

Should be Supplied to NHS Within Weeks

June Raine, MHRA chief executive, said in a press release: "It’s very good news that after a rigorous review, we have approved Kaftrio for children aged 6-11, enabling the NHS to bring this life-saving treatment to more children suffering from cystic fibrosis." 

After the decision, the new doses of Kaftrio should be supplied to the NHS in England within weeks, enabling clinicians to prescribe to all young CF patients aged 6-11 where it is considered appropriate. Access to Kaftrio is also included in long-term agreements in place between Vertex and the NHS in Scotland and Wales.

The National Institute for Health and Care Excellence (NICE) will continue to collect data on Kaftrio from the UK Cystic Fibrosis Registry to inform a future health technology appraisal. An interim summary report is expected in February this year, with a final review in June 2023. 

"This approval is an important milestone in our efforts to bring medicines to people with CF as early in life as possible," said Dr Andrew Wilfin, UK & Ireland Medical Director at Vertex. "More than 400 children in Great Britain are now eligible for treatment for the first time, bringing us another step closer to our goal of providing a treatment option to all people with this devastating disease."

The Cystic Fibrosis Trust said it was "delighted at the positive impact this will have for many children and their families", and welcomed the licence extension as "an important next step in limiting lung damage and slowing long term decline in health".

David Ramsden, chief executive at the Trust, said: "This means that now over 1500 children across the UK and Northern Ireland stand to benefit from this drug and limit the damage CF does in these critical early years."

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