HSE approval of cystic fibrosis drug hailed as 'wonderful Christmas present' for patients

HSE approval of cystic fibrosis drug hailed as 'wonderful Christmas present' for patients
File photo of a girl with cystic fibrosis

The first drug to treat the underlying cause of cystic fibrosis (CF) for around 80% of patients in Ireland has been approved by the HSE and is being considered for approval by the European Medicines Agency (EMA).

Trikafta is the first approved treatment that is effective for CF patients aged 12 years and over.

All Irish CF patients over 12 with at least one copy of the F508del gene mutation will get access to Trikafta if it is approved by the EMA. A decision from the EMA is expected in the first quarter of 2020.

The HSE expanded its existing reimbursement agreement with Vertex Pharmaceuticals, which manufactures the drug. Vertex submitted a marketing authorisation application to the EMA on October 31.

"With this expanded agreement, even more patients in the Republic of Ireland will be among the first in Europe to benefit from the triple combination treatment once the medicine is licensed," said Ludovic Fenaux, Senior Vice President of Vertex International.

Philip Watt, the CEO of Cystic Fibrosis Ireland welcomed the HSE's approval of the groundbreaking treatment.

Philip Watt, Cystic Fibrosis Ireland
Philip Watt, Cystic Fibrosis Ireland

"This is a most wonderful Christmas present for all those with cystic fibrosis in Ireland," Mr Watt said.

"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."

"This is a new dawn for CF care in Ireland and we thank Minister for Health Simon Harris and the Irish Government for their care and compassion," Mr Watt said.

"The decision today 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 cystic fibrosis in Ireland will increase steadily over the next few years and, indeed, Ireland may overtake other countries."

Trikafta was approved in the United States of America on 21 October 2019, some five months ahead of schedule.

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