GENERAL MEDICINE
New drug therapy approved for cystic fibrosis
December 16, 2019
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The news that a breakthrough drug therapy is to be made available to people with cystic fibrosis (CF) in Ireland has been widely welcomed.
The HSE has confirmed that following discussions with the pharmaceutical company, Vertex, it has expanded its existing reimbursement agreement, so that all CF patients eligible to be prescribed the drug therapy known as Trikafta, "will have access subject to receipt of European Medicines Agency (EMA) approval".
Trikafta is a combination drug therapy that is made up of three drugs, Elexacaftor, Tezacaftor and Ivacaftor. It is the first approved treatment that is effective for CF patients who are over the age of 12, with at least one F508del mutation.
This affects 90% of the worldwide CF population and 80% of the Irish CF population, which is around 1,000 people here.
Trikafta was approved in the US in October, some five months ahead of schedule. Now, because of the HSE's announcement, all patients in Ireland with CF who are over the age of 12, and who have at least one copy of the F508del gene mutation, will get access to this drug therapy as soon as it is approved by the EMA.
This decision is expected in the first quarter of 2020.
"This is a most wonderful Christmas present for all those with CF in Ireland. The drug therapy Trikafta is what many scientists and patients have been waiting for since the basic genetic cause of CF became understood in 1989," explained Philip Watt, CEO of Cystic Fibrosis Ireland (CFI).
He said that once approved, it is likely that most CF patients on existing drugs, such as Orkambi, will transfer over to Trikafta depending on their advice of their doctors.
"One clinical trial showed that Trikafta increased lung function by an average of 14%. Research also shows a decrease in exacerbations by up to 60%. This is a new dawn for CF care in Ireland," Mr Watt insisted.
He pointed out that this decision "effectively means Ireland will be the first country in Europe to receive this drug for its patients".
"We have great hope now that the life expectancy of people with CF in Ireland will increase steadily over the next few years and, indeed, Ireland may overtake other countries," he added.
CF is Ireland's most common life-threatening inherited disease. There are around 1,200 people in Ireland affected.
It affects the regulation of absorption and secretion of salt and water in various parts of the body including the lungs, pancreas and gastrointestinal tracts. This defect inhibits the flow of salt and water through the body's cells, causing a build-up of thick, sticky mucus, which can clog airways and harbour harmful bacteria.
For more information on the condition and CFI, click here.