Around 70 very young children are to be offered the cystic fibrosis drug Orkambi, it emerged today.
Vertex Pharmaceuticals said the treatment will be available for suitable children aged two to five years.
These children woud have two copies of the F508del mutation, the most common form of the disease.
Under a deal reached between manufacturer Vertex Pharmaceuticals and the HSE and Government in 2017, new treatments and extensions of existing treatments to new patients are made available to Irish patients.
The drug was made available to six to 11-year-olds from March 2018.
Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex said: ‘‘By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes.’’
Accordingly, Vertex will work closely with the HSE and relevant officials to secure timely access for these patients, and we expect it will be made available shortly.
It is estimated that there are around 70 patients aged 2-5 who are eligible for this treatment in Ireland.