Delight with Cystic Fibrosis drug approval

A CASTLEDERG man who has campaigned for years to have life-changing Cystic Fibrosis drugs made available has said his dreams have come true.
Liam McHugh has delightfully welcomed the news that the Kaftrio drug is one step closer to being widely available in the North.
Trikafta, now named Kaftrio in Europe, is the first drug to treat “the underlying cause of the disease” of approximately 90 per-cent of patients.
In recent days, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) gave the drug the ‘thumbs up’, meaning it could be just months away from total approval.
Liam’s daughter Rachel McHugh had her life transformed after she was initially given Orkambi as part of a clinical trial in 2013.
Rachel remains on the drug to this day, but she will also be able to avail of the new drug.
Liam McHugh believes this drug is going to save thousands of lives, and transform the lives of Cystic Fibrosis sufferers.
“This is the stuff we have been dreaming about for years,” said Liam.
“We never thought we would see it happen and it still hasn’t sunk in.
“This drug was already available in America, and it had been given out here on compassionate grounds.
“Orkambi and Symkevi have been keeping people well for now, but this is going to change people’s lives.
“This drug will be suitable for 90 per-cent of sufferers and it is a million times more effective than the other drugs.
“Young children with Cystic Fibrosis will be able to live a full life, and for people like Rachel who have been well through Orkambi, this drug is going to be mind-blowing,” said Liam.
The deal has been agreed by the NHS England, but a tag along clause in the contract allows Northern Ireland to sign up for the drug.
While the official announcement from health officials has not yet been made, Liam is hopeful that it is imminent.
He said, “I have been torturing their office and have not received a reply but we are confident that they are working at it and an announcement is expected.
“It will be the biggest day in Cystic Fibrosis history.
“For 18 years, there was no progress, but the past ten years has been incredible for us,” said Liam.
However, while Liam is delighted with the progress, he hasn’t forgot about the ten per-cent of Cystic Fibrosis sufferers who cannot avail of this drug.
Liam continued, “There are around 1,800 mutations of Cystic Fibrosis so it is incredible that they have managed to find a drug for 90 per-cent of them.
“There are still people out there who cannot get this drug, and it must be very hard for them to watch this.
“We cannot forget about them and we will continue to fight for them to receive treatment,” concluded Liam.

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