EMA panel recommends Kaftrio for range of CF-causing mutations
Vertex says expanded label would make 'thousands' eligible for treatment
A European Medicines Agency (EMA) committee recommended that the label for Kaftrio be expanded to cover treatment for people with cystic fibrosis (CF) caused by a wider array of mutations, Kaftrio’s developer Vertex Pharmaceuticals said.
The Committee for Medicinal Products for Human Use (CHMP) opinion will be reviewed by the European Commission, which has final say over medication approvals in the European Union.
“If approved, thousands of additional patients across Europe will be eligible for a CFTR modulator, bringing us closer than ever to our goal of getting treatments that address the underlying cause of the disease to all people living with CF,” Fosca De Iorio, MD, vice president of international medical affairs at Vertex, said in a company press release.
Expanding Kaftrio beyond most common mutation
CF is caused by mutations in the gene that provides instructions to make the CFTR protein. Kaftrio, sold as Trikafta in the U.S., contains a combination of three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor), which are oral therapies that can boost the activity of this protein in people carrying certain CF-causing mutations.
By boosting the activity of the mutated protein, the modulator therapy has been proven to help ease CF symptoms in patients with responsive mutations.
Kaftrio is approved in Europe in combination with Kalydeco (ivacaftor) to treat people with CF ages 2 and older who carry at least one copy of F508del, the most common CF-causing mutation. The CHMP recommended that the approval be expanded to cover anyone with CF who carries a non-class 1 mutation.
Class 1 mutations result in little to no CFTR protein being produced. Since CFTR modulators work by targeting the CFTR protein, these medications are not expected to provide any benefit for patients with mutations that prevent the protein from being made. The CHMP recommendation would effectively mean that Kaftrio plus Kalydeco would be approved for anyone with CF who can produce at least some CFTR protein, regardless of how the protein is mutated.
“We are thrilled by the positive CHMP opinion in support of expanding the Kaftrio in combination with ivacaftor indication to include all CF patients 2 years and older who make CFTR protein,” De Iorio said.
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