Hailey’s joy after she wins battle for wonder drug that may save her life..
Cystic fibrosis patient Hailey given miracle drug that may save her life after heartbreaking appeal in the
A YOUNG cystic fibrosis patient was “on cloud nine” last night after doctors told her she was to get the miracle drug which could save her life.
Just three days after the Daily Record told how Hailey Mann, 24, needed Kaftrio to give her any hope of survival, drug firm Vertex told doctors they had shipped the drug to her.
A delighted Hailey, from Aberdeenshire, said: “It is unbelievable. Thank you for everything.
“If it wasn’t for all the pushing by the Daily Record it wouldn’t have happened. You kicked them into gear.”
On Tuesday, we told how Hailey had been told to start planning her end-of-life care after her lung function fell to just 14 per cent. In order to be put on the lung transplant list, she needs to put on more weight – and a complication of cystic fibrosis is that patients find weight gain difficult.
Kaftrio, knowns as Trikafta in the US, helps with weight gain, digestive problems and lung function, so Hailey was desperate to get it.
It is her only hope of living long enough to get a transplant.
The drug has had remarkable results.
Scots teenager
Anna Pignatelli was unable to walk up stairs at school in March. But after she was given the drug on compassionate grounds, she is now on the Couch to 5k running plan. Hailey said: “I can’t even imagine these things.” Hailey was told in January that getting Kaftrio on compassionate grounds was an option but it took until this week for the process to get moving. The Scottish Government announced earlier this week that it had struck a deal with Vertex to provide the drug on the NHS.
But it cannot be prescribed until it receives its European Medicines Agency licence, which will still be a few weeks away – and could have come too late to save Hailey.
Her only hope remained to get the drug from Vertex on compassionate grounds.
For the past few days the Record has been speaking to the drug company, emphasising how urgently Hailey requires the drug. The company said the decision to supply drugs on compassionate grounds are always “difficult and complex” but last night they came through for Hailey.
She said: “All the forms were completed and sent off and then this afternoon I got a phone call when I was in the bath. The doctor said he couldn’t speak to my mum, it had to be me.
“When I got to the phone he told me the drugs have now been shipped. They are on their way.
“I am on cloud nine. I can barely speak. They should arrive in Aberdeen in three to five days. I can’t get my head round it. I honestly can’t believe it.
“The weekend I am going to have is unreal. What an amazing start to the weekend.
“But I feel my legs are like jelly. Everybody has been waiting on this news and I am looking forward to calling to tell them.
“All these months I have been waiting and in less than a week I should be starting to take them. It is unbelievable.”