‘Lifeline’ cystic fibrosis drug is approved for more children but Wakefield mum says her son’s future is still uncertain

Access to a ‘miracle drug’ that can help people with cystic fibrosis live longer and fuller lives has been granted for more children after its future was thrown into doubt.
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A decision was due over whether Kaftrio would be approved for children between the ages of two and six but draft guidance from the National Institute for Clinical Excellent (NICE) raised concerns it would be deemed too expensive.

Wakefield mum Kirsty Ginnelly said the earlier guidance could mean her son, Oscar, one, would miss out on the “lifeline” treatment.

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Now, manufacturer Vertex Pharmaceuticals has confirmed approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) for Kaftrio for children with cystic fibrosis (CF) ages two to less than six years and Kalydeco for infants aged one to four months who have specific, relevant mutations as part of their condition.

Kirsty Ginnelly hopes her son Oscar will have access to Kaftrio.Kirsty Ginnelly hopes her son Oscar will have access to Kaftrio.
Kirsty Ginnelly hopes her son Oscar will have access to Kaftrio.
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Treatment to help Wakefield baby with cystic fibrosis live a longer and ‘normal’...

Dr Andrew Wilfin, senior medical director at Vertex UK&I, said: “Both approvals are important milestones for eligible people living with CF, as more children are now eligible for a treatment that targets the underlying cause of their disease.

"These milestones reaffirm our ongoing commitment to providing a treatment option to all people with CF no matter what their genotype or age.”

Kirsty welcomed the decision to approve the drug but said Oscar’s future is still not guaranteed.

Oscar with mum Kirsty and sister PippaOscar with mum Kirsty and sister Pippa
Oscar with mum Kirsty and sister Pippa
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She said: “This is amazing news for children who will now be able to access the modulators.

“Although age eligibility has decreased this still means that children under the age of two and babies yet to be born could still lose access to the modulators. This includes Oscar.

“Imagine watching others with cystic fibrosis thrive whilst your disease progresses because you were born too late.

“Although things are moving in the right direction there is still uncertainty for many toddlers, babies and parents across the UK.

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"We need to ensure that life-saving medication is available for everyone eligible. No child should miss out on the chance of a future because of their age.”

A person with cystic fibrosis taking Kaftrio could live until around 80.

The NHS says currently, about half of people with cystic fibrosis will live past the age of 40.

The draft guidance said people with cystic fibrosis who were already using the treatment would continue.

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