HSE approves 'groundbreaking' cystic fibrosis drug for children aged 6-11

A demonstration at Leinster House last September calling for the availability of Kaftrio for children with cystic fibrosis. File picture: Shane O'Neill

The families of 35 children with cystic fibrosis are celebrating after the drug Kaftrio was approved by the HSE for use by children aged between 6-11.

The drug had previously been excluded from the children within this age bracket. It has now been extended for use following lengthy engagements with the drug manufacturer Vertex.

Cystic Fibrosis Ireland warmly welcomed the decision to allow the children access to the therapy, which they have been denied for almost a year.

The organisation used today's announcement to urge "future-proofing" and ensure that when the drug is extended to children aged 2-5 years, disputes such as this can be avoided.

CEO of Cystic Fibrosis Ireland, Phillip Watt welcomed the decision, saying: “In quietly celebrating with all who have contributed to this positive outcome today, we particularly think of, and thank, all of the parents, friends and families of the 35 impacted children directly affected by this dispute. 

"Their voice, their dignity and their strength over the past year has been the key factor in providing the necessary momentum for resolving a crisis that should never have occurred in the first instance. 

"It is an honour for Cystic Fibrosis Ireland to continue to work with, and support, the Kaftrio 35 as best we can."

HSE Chief Clinical Officer Colm Henry delivered the announcement, adding: "We have finalised the negotiated agreement with the manufacturer and are pleased to announce that the HSE has now approved this drug which will improve the lives of the children who can now avail of it."

The drug is used for treatment of cystic fibrosis patients who are heterozygous for the F508del mutation and either a minimal function (MF) mutation, or an unknown mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Health Minister Stephen Donnelly welcomed the introduction of the "life-changing drug".

"This is good news for the young children with cystic fibrosis who can now access this therapy. It is another important milestone that will help us to continue to improve outcomes for patients with Cystic Fibrosis in this country,” Mr Donnelly said.