Miracle drug needed for ‘Celtic gene’ cystic fibrosis victims in Ireland

Govt urged to buy drug that costs $300,000 a year for genetic lung disease

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Photographer Kyle Monk's image depicting the sad reality of cystic fibrosis - the degenerative lung disease has its highest figures in Ireland

Photo by Kyle Monk

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The Health Service Executive (HSE) in Ireland is being urged to approve a revolutionary new drug, that will benefit certain sufferers of cystic fibrosis.

The new drug, called Kalydeco, treats the G551D mutation – nicknamed the ‘Celtic gene’. Up to 1,100 people in Europe and 100 in Ireland possess the ‘Celtic gene.'

It is incredibly expensive, close to $300,000 for a year’s supply, which makes it imperative that governments buy it.

“Kalydeco is one of the most important advances in the treatment of cystic fibrosis since the discovery of the CF gene in 1989. By treating the underlying cause of the disease in people with the G551D mutation, Kalydeco helped them breathe more easily, gain weight and resulted in certain improvements in quality of life, "Stuart Elborn, MD, Kalydeco investigator and President of the European Cystic Fibrosis Society said.

It has already been approved by the drug regulators in the US and Europe, but Irish health regulators have yet to give it the go ahead.

Manufactured by Vertex, the drug treats people with cystic fibrosis aged six and over who have at least one copy of the G551d mutation.

Read more: New Northern Irish drug to target ‘Celtic Gene’ - new hope for Cystic Fibrosis patients

Ireland has the highest proportion of people with CF in the world. According to the Cystic Fibrosis Association of Ireland about 50 new cases of cystic fibrosis are diagnosed each year.

Now regulators in Ireland must approve the drug, ahead of pricing negotiations between Vertex and the government’s pharmaceutical unit.

Councillor David McGuinness told TheJournal.ie it is now “time for Ireland to lead the way in treating this difficult illness”.

“Across Europe, and the rest of the world, campaigners for those with Cystic Fibrosis will be lobbying politicians and medical organisations to incorporate Kalydeco into their cystic-fibrosis treatment plans and it is time for Ireland to lead the way in treating this difficult illness which affects so many in Ireland,” said McGuinness.

“Ironically, the particular strain of CF which Kalydeco treats is referred to as the ‘celtic’ gene. Ireland has high rates of CF given its population and some commentary, on this topic, attribute this to the famine when higher percentages of the population with CF survived starvation given their ability to endure severe hardship. Nowadays, Ireland has many families who suffer from CF and it is now time for Minister Reilly and the HSE to support these families by endorsing this drug”, he added.

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