Orkambi is a therapy that combines ivacaftor and lumacaftor to treat cystic fibrosis patients who have two copies of the mutated F508del gene. The oral CFTR modulators work by amending the CTFR protein that is faulty (due to the mutation in the gene), working to the root cause of the problem rather than just treating the symptoms of the disease.
Since its FDA approval in July 2015, the drug has helped thousands of cystic fibrosis sufferers in the U.S. and other countries around the world. However, cystic fibrosis patients in Ireland have been unable to receive Orkambi. The government hadn’t approved the drug because of the costs associated with it.
According to the Irish Examiner, Ireland has the highest number of cystic fibrosis patients per capita. Those living in the country are three times more likely to be born with the disease than people in the U.S.
Cystic fibrosis patients and their families have been campaigning for funding for the treatment through television appearances and protests. Sadly for some prominent members of Ireland’s CF community, their wish was granted too late.
The Irish medical board has finally come to an agreement with Vertex, the makers of Orkambi. Cystic fibrosis patients are now able to take advantage of this life-changing drug.
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