Kaftrio approved in Europe for wider range of CF mutations
Label expansion makes about 4,000 patients in EU newly eligible for treatment
The European Commission has approved a label expansion for Kaftrio in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) caused by a wider range of mutations.
The decision follows a positive endorsement by the Committee for Medicinal Products for Human Use, a branch of the European Medicines Agency. With the broader indication, about 4,000 people with CF in the European Union are newly eligible for a disease-modifying therapy.
Due to existing reimbursement agreements in Austria, Denmark, Ireland, Norway, and Sweden, and access provisions in certain healthcare systems, such as Germany, the Kaftrio plus Kalydeco combination will soon be available to eligible patients in those countries.
“We have been working for years to bring treatment options to all people with cystic fibrosis, including those with ultra rare mutations,” Carmen Bozic, MD, executive vice president of global medicines development and medical affairs, and chief medical officer at Vertex Pharmaceuticals, which developed Kaftrio, said in a company press release.
In CF, genetic mutations cause a deficiency or malfunction of the CFTR protein, which plays a crucial role in regulating the balance of salt and water on cell surfaces. Without proper CFTR function, mucus on these surfaces becomes unusually thick and sticky, leading to buildup in various organs, especially the lungs, pancreas, intestines, and liver, and resulting in a range of symptoms.
Kaftrio, sold as Trikafta in US, contains 3 CFTR modulators
Kaftrio, sold as Trikafta in the U.S., contains three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor). These oral therapies are designed to boost the activity of CFTR in people with certain CF-causing mutations.
The Kaftrio-Kalydeco combination is approved in Europe for people with CF, 2 and older, who carry at least one copy of F508del, the most common CF-causing mutation, in the CFTR gene.
The therapy’s indication has now been expanded to cover CF patients with a non-class 1 mutation, which results in little to no CFTR protein being made. Although CFTR modulators are not expected to benefit patients with mutations that prevent the protein from being made, this label expansion means that Kaftrio plus Kalydeco is approved for anyone with CF who can produce at least some CFTR protein, regardless of the mutation.
“We are pleased that the European Commission has broadened the Kaftrio indication to include all CF patients 2 years and older who have at least one non-class 1 mutation, ensuring that even more people living with CF can benefit from this transformative medicine,” Bozic said.
Vertex said it will continue to collaborate with reimbursement authorities across the European Union to ensure access for all eligible patients as soon as possible.
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