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CHMP Favors Kaftrio to Treat CF Patients in EU Starting at Age 12

A European Medicines Agency (EMA) committee favors extending the label for Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to treat cystic fibrosis (CF) patients 12 and older who have at least one copy of the CFTR gene with an F508del mutation.

The European Commission (EC) approved the same combination last year to treat people 12 and older whose CF is due to either two F508del mutations or one F508del mutation and one minimal function mutation in the CFTR gene. This approval did not cover patients with one F508del mutation and one of several other types of mutations.

If the EC accepts the opinion of the EMA’s Committee for Medicinal Products for Human Use (CHMP), Kaftrio would be available for the majority of people with CF in Europe. Historically, the EC accepts most CHMP recommendations.

“Today’s opinion is an important step towards bringing this medicine to any patient with at least one F508del mutation, including those with a gating or residual function mutation who were not previously eligible for the triple combination therapy,” Nia Tatsis, PhD, executive vice president, chief regulatory and quality officer at Vertex Pharmaceuticals, which markets Kaftrio, said in a press release.

Everyone has two copies of the gene CFTR (cystic fibrosis transmembrane conductance regulator), one inherited from each biological parent. Mutations in this gene lead to the production of a CFTR protein that does not work correctly. Different types of mutations in the gene have different effects on the functionality of the protein.

F508del is the most common CF-causing mutation, and an estimated 90% of CF patients have at least one F508del mutation.

Kaftrio, which is sold as Trikafta in the U.S., contains a combination of three CFTR modulators — medications that make the CFTR protein work more effectively. Kaftrio is used in combination with Kalydeco, another CFTR modulator marketed by Vertex.

The CHMP’s opinion was based on data from a global Phase 3 clinical trial (NCT04058353) sponsored by Vertex. The trial enrolled 271 patients ages 12 and older with one F508del mutation and one other mutation: either a gating mutation (meaning part of the CFTR protein gets “stuck closed”), or a residual function mutation (meaning not enough working CFTR protein gets to the cell’s surface).

Results demonstrated that treatment with Kaftrio led to clinically and statistically significant improvements in lung function and significantly reduced sweat chloride levels.


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