Orkambi extended to treat CF patients in EU starting at age 1
Eligible children are those with two F508del mutations in CFTR gene
The European Commission approved expanding the use of Orkambi (lumacaftor/ivacaftor) to include children as young as 1 year old with cystic fibrosis (CF) and two copies of the F508del mutation in the CFTR gene.
Vertex Pharmaceuticals’ therapy was first approved in the European Union (EU) in 2015 for CF patients ages 12 and older with two F508del mutations — the most common CF-causing mutation. That label was extended to children ages 6-11 in 2018 and to those ages 2 to 5 the next year.
“This approval will offer some of the youngest children with cystic fibrosis the chance of improved outcomes, by treating their disease at a young age,” Carmen Bozic, MD, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, said in a company press release.
“With this important milestone, we move ever closer to our goal of providing medicines that treat the underlying cause of CF to all people living with the disease,” Bozic added.
About 300 toddlers with CF in European Union likely eligible for Orkambi
The regulatory decision, which is estimated to affect nearly 300 eligible children, comes about two months after the Committee for Medicinal Products for Human Use, a branch of the European Medicines Agency, issued a positive recommendation on the label expansion. While the commission’s final decision does not have to be in line with that opinion, it often is.
For European countries with existing reimbursement agreements or other healthcare provisions, including Austria, Denmark, the Republic of Ireland, Sweden, and Germany, this clearance means that patients ages 1-2 will have access to the therapy almost immediately. It is already available to such patients in the U.K.
Vertex says it will continue to work with reimbursement bodies across the EU, Australia, and Canada to ensure access for all eligible patients.
In CF, mutations in the CFTR gene lead to a lack of a working CFTR channel protein that’s involved in moving salts and water in and out of cells. The consequent production of thick, sticky mucus in organs drives disease symptoms, which are usually evident soon after birth.
“CF symptoms and organ damage can manifest very early in life, so it is crucial to start treatment as early as possible,” said Silvia Gartner, MD, a specialist in pediatrics and pneumonology, and coordinator of the Pediatric Cystic Fibrosis Center in Barcelona, Spain.
Orkambi is an oral therapy containing the CFTR modulators ivacaftor and lumacaftor. Lumacaftor is a CFTR corrector that helps the CFTR protein take on the right shape, allowing more of it to reach the cell’s surface, whereas ivacaftor is a CFTR potentiator, helping to hold the channel protein open and enable more normal salt flow.
Together, they aim to increase overall CFTR protein function, preventing the buildup of thick and sticky mucus.
Orkambi’s expanded approval for younger children was backed by data from a Vertex-sponsored Phase 3 clinical trial (NCT03601637), which tested Orkambi in children ages 1 and up to 2 with two copies of the F508del mutation. Participants were treated for 24 weeks, or about six months.
Results indicated the treatment was generally safe and well tolerated, with a pharmacological profile similar to that seen in older patients. Its use also lowered patients’ sweat chloride concentration, an indicator of improved CFTR function.
“Today’s approval provides us with a medicine that gives a window of opportunity to possibly delay the onset of CF for these very young eligible children,” Gartner said.