US Approves New CF Triple Combination Drug Five Months Ahead of Schedule

‘Trikafta‘ offers significant further hope for people with CF in Ireland.  

The US Food and Drug Administration (FDA) has approved the first triple combination therapy to treat patients with the most common cystic fibrosis (CF) gene mutation worldwide. 

Vertex Pharmaceuticals' new drug is called Trikafta and combines elexacaftor, ivacaftor, and tezacaftor and is approved for patients age 12 years or older with CF and at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. This gene mutation impacts on 73 per cent of the CF population in Ireland. 

Philip Watt CEO of CFI stated

"Under the innovative drug pipeline deal agreed between Vertex and the Irish Government in 2017, when Orkambi was approved, most people with CF in Ireland aged 12 and over will be eligible for this very promising new CF drug therapy ‘Trikafta’ once it is approved by the European Medicines Agency. CFI urges the EMA to fast-track this drug therapy as soon as possible to enable it to become available in Ireland. Once it becomes available in Ireland, Trikafta will likely supersede existing CF drugs Orkambi (and Symkevi)for many CF patients In Ireland. However this should not underestimate the value of Orkambi which has acted as an important bridging medication and has resulted in a significant decrease in hospitalisations for many CF patients in Ireland and over 70 percent reduction in the use of  expensive anti-biotics. This is a very exciting time in the history of CF worldwide and in Ireland, but it should not gloss over that CF remains a very challenging disease, and the need for 20 CF in patient rooms in Beaumont Hospital."

Read the full statement on the FDA approval of Trikafta here.