A four-year-old with cystic fibrosis has received the Christmas gift of her dreams – a wonder drug for her crippling condition.
Ayda Louden’s family, from Carlisle, Cumbria, spent years calling for NHS access to Orkambi. It was finally approved in October.
The schoolgirl got her first delivery of the life-changing drug, which should extend her life by decades, two weeks ago.
Her engineer father Dave said: ‘What better gift for Christmas than the gift of your daughter living a healthier, longer life?
Ayda Louden got her first delivery of the life-changing drug, which should extend her life by decades, two weeks ago
Her family spent years calling for NHS access to Orkambi. It was finally approved in October (Ayda is pictured with her brother Alfie, mother Stacey and father Dave)
‘The delivery driver was greeted by the whole family, honestly I could have hugged him, but went for a handshake instead.’
Orkambi, made by Boston-based Vertex, is said to improve lung function and reduce breathing difficulties.
Before the deal with NHS bosses was struck, the pharmaceutical rejected an offer of £500million over five years for access to the drug.
It is unclear what the final agreed price was. Campaigners said it was a ‘very special day’ for patients.
Mr Louden, 35, and wife Stacey, 32, who also have an eight-year-old son called Alfie, have spent most of Ayda’s life campaigning for it to be made available on the NHS.
After hearing the good news in October the family attended a CF clinic in November to find out about their daughter’s treatment going forward.
Mr Louden, 35, and wife Stacey, 32, who also have an eight-year-old son called Alfie, have spent most of Ayda’s life campaigning for it to be made available on the NHS
Mr Louden said it was an ‘amazing feeling’ to see the excitement of Ayda’s medical team that they finally had the best tools to fight the condition
WHAT IS CYSTIC FIBROSIS?
Cystic fibrosis is an incurable genetic disease that affects around 70,000 people worldwide.
A 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.
Source: Cystic Fibrosis Foundation
Mr Louden said: ‘It certainly is a day I will never forget… Words cannot really describe the emotions of that day – pure joy.’
He said it was an ‘amazing feeling’ to see the excitement of Ayda’s medical team that they finally had the best tools to fight the condition.
Ayda began the twice a day treatment, which is a combination of drugs lumacaftor and ivacaftor, on December 14.
Mr Louden said: ‘Ayda has taken to Orkambi really well and has had no negative side effects so far.
‘She has been really brave and mature beyond her years, it is not a nice tasting medicine.
‘But she’s put that to one side and has taken it every morning and night with minimal complaints.’
He added: ‘She seems to realise its important which is really good, we are so proud of her.’
Ayda’s parents say the treatment arrived just in time because cystic fibrosis patients are at the highest risk of infection over winter.
One of Ayda’s most severe symptoms over the years has been weight loss, however, it’s hoped her new treatment will see that improve.
Mr Louden said Orkambi will help keep her lungs healthier for longer, and reduce the amount infections, hospital stays and irreversible lung damage.
Mr Louden said: ‘Ayda has taken to Orkambi well and has had no negative side effects so far’
Both her parents are aware the treatment will not cure the cystic fibrosis but it gives them a boost and more energy as a family to fight
He added: ‘All of this will increase her life expectancy by decades and giver her a better quality of life.
‘It’s not just Ayda Orkambi has had an effect on, as a family its a weight lifted to know Ayda now has access to the best possible treatment.
‘Her brother is especially relieved she now has Orkambi as he begins to understand her condition more and how it will help his sister.’
Both her parents are aware the treatment will not cure the cystic fibrosis but it gives them a boost and more energy as a family to fight.
Mr Louden added: ‘We are so grateful that Orkambi has finally been made available on our wonderful NHS.
‘But four years was an unacceptable amount of time to wait, and lessons must be learned in the future.
‘Sadly hundreds of cystic fibrosis lives were needlessly lost during the wait for Orkambi.’
Cystic fibrosis is a debilitating illness, which creates a mucus build-up that causes chronic lung infections and progressive lung damage.
The incurable disease affects around 70,000 people worldwide, including 3,000 in the UK, according to figures.
Ayda’s parents say the treatment arrived just in time because cystic fibrosis patients are at the highest risk of infection over winter
WHAT IS ORKAMBI?
Orkambi is a medication licensed for use by people in the UK with cystic fibrosis, but it is not offered on the NHS except in extreme circumstances.
The drug targets the F508del gene mutation, which affects about 50 per cent of all people with cystic fibrosis.
It is made of a combination of drugs – lumacaftor and ivacaftor – which work together to keep a healthy balance of salt and water in the lungs.
There may be more than 4,000 people with life-limiting cystic fibrosis in the UK who could benefit from the drug.
But the National Institute for Health and Care Excellence (Nice) said in 2016 may not be cost-effective enough, at £104,000 per patient per year, to be offered on the NHS.
More than 117,000 people signed a petition calling on the Government to make Orkambi available on the NHS, and Parliament discussed the petition in March 2019.
It was finally made available on the NHS in England on October 24 after four-years of negotiations.