Derry mum's joy as medicines deal means infant daughter will enjoy 'longer, better life'

A Derry mum has said she is "overwhelmed and shocked" by news that a breakthrough new cystic fibrosis medicine will be available for her infant daughter.

Life-changing cystic fibrosis medicines are now set to be available to patients in Northern Ireland after a US pharmaceutical company confirms it has struck a deal with health authorities on the cost of the drugs.

Derry mum-of-two Laya Barr, speaking to Belfast Live, said that if the medicine works as expected for her daughter Robyn - who recently celebrated her first birthday - it will mean "a longer life, less visits to hospital, and a better life".

Read more: Derry mum in last ditch plea over cystic fibrosis medicines deemed 'too expensive'

Read more: Newtownabbey mum hits out at U-turn on cystic fibrosis wonder drug that saved her life

Families have been campaigning for access to a trio of breakthrough drugs known as Kaftrio, Symkevi and Orkambi that were deemed too expensive for the NHS to pay for back in November.

But the US pharmaceutical company Vertex has now confirmed, in a statement issued to Belfast Live on Tuesday, that "reimbursement agreements" have now been reached with health authorities in both Northern Ireland and Wales to allow patients here to have "extended, long-term access".

The news will be welcomed by families across Northern Ireland who have campaigned for a deal to be reached.

An appraisal by the UK's National Institute for Health and Care Excellence (NICE) that started in 2022 found that, while clinically effective, the cost was too high for the NHS to pick up the tab.

That changed last week when NICE gave approval for the life-changing cystic fibrosis modulator drugs to be made available on the NHS in England.

And the company behind the drugs, known as Vertex, has now confirmed a deal has been struck with health authorities in both Northern Ireland and Wales.

Laya Barr, who was among those campaigning for the medicines to be made available here, said she was "completely overwhelmed and shocked" by the good news.

"The amount of work we had put in to get people to sign the petition has actually helped massively," she said. "It is unreal. It is something we had really worked for."

Asked what access to the medicines, known as cystic fibrosis modulator drugs, will mean for her daughter Robyn, she said: "It means she is going to be able to live a longer life, less admissions to hospital, a better life. It has just made the biggest difference for us."

A spokesperson for Vertex said: "We are pleased to confirm that we have finalised the reimbursement agreements with NHS Wales and NHS Northern Ireland for extended long-term access to cystic fibrosis (CF) medicines KAFTRIO® (ivacaftor/tezacaftor/elexacaftor), SYMKEVI® (tezacaftor/ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor).

"The agreements are comparable to the NHS England agreement and provide access to these medicines for all existing and future eligible CF patients in Wales and Northern Ireland.

"They also include access to any future licence extensions of these medicines and a commitment by all parties to work together on a path towards rapid access for all eligible patients for the next in class triple combination treatment for CF, the investigational therapy vanzacaftor/tezacaftor/deutivacaftor, subject to authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA)."

The spokesperson added: "We continue to work closely with the NHS authorities in Scotland to finalise this agreement as soon as possible."

For all the latest news, visit the Belfast Live homepage here and sign up to our daily newsletter here.