European Commission extends approval of cystic fibrosis medicine

For the first time, a medicine to treat the most common form of cystic fibrosis has been approved in paediatric patients aged one to under two years old.

European Commission extends approval of cystic fibrosis medicine

European Commission (EC) has granted approval for the label extension of cystic fibrosis medicine ORKAMBI® (lumacaftor/ivacaftor) for children aged one to <2 years old.

The oral treatment is indicated to treat these individuals with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene for the first time. In these patients, the CFTR protein is not processed and trafficked normally within the cell. The result is little to no CFTR protein at the cell surface.

How does the oral cystic fibrosis medicine work?

Ultimately, the combined actions of lumacaftor and ivacaftor help to hydrate and clear mucus from the airways.

Small molecule drug lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein. Ivacaftor, another small molecule CFTR potentiator, helps CFTR proteins to transport salt and water across the cell membrane.

Dual cystic fibrosis modulator therapy shows efficacy in four-year study

Notably, the approval means there is now a medicine available offering “a window of opportunity to possibly delay the onset of cystic fibrosis for these very young eligible children,” commented Dr Silvia Gartner, Specialist in Pediatrics and Pneumonology, Coordinator of the Pediatric Cystic Fibrosis Center, Barcelona.

As “cystic fibrosis symptoms and organ damage can manifest very early in life… [it is important] to start treatment as early as possible,” explained Dr Gartner.

Therefore, the European Commission’s regulatory approval is an “important milestone” according to Dr Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex Pharmaceuticals.

Expanding access to ORKAMBI (lumacaftor/ivacaftor)

In the UK, children aged one to <2 years old have had access to this expanded indication for ORKAMBI since the Medicines and Healthcare products Regulatory Agency (MHRA) approval in March 2023. This is due to long-term reimbursement agreements in these regions.

Eligible patients in countries such as Austria, Denmark, the Republic of Ireland and Sweden will have access to the expanded indication for the cystic fibrosis medicine shortly following regulatory approval by the EC, due to similar regulatory agreements.