EU, UK Considering Expanding Kaftrio to Children Ages 6 and Older

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by Forest Ray PhD |

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Kaftrio and children

Regulatory agencies in the European Union and in the U.K agreed to consider expanding the use of Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to children with cystic fibrosis (CF) who have at least one F508del mutation starting at age 6.

The European Medicines Agency (EMA) and the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) validated an application from Vertex Pharmaceuticals, the treatments’ maker.  The MHRA and the EU Committee for Medicinal Products for Human Use will now review and decide on the request separately.

“Today’s news is an important milestone in broadening our access worldwide and we are pleased that we were able to file with the EMA and the MHRA in parallel,” Nia Tatsis, PhD, Vertex’s executive vice president, chief regulatory and quality officer, said in a press release.

F508del is the most common disease-causing mutation of the CFTR (cystic fibrosis transmembrane conductance regulator) gene, occurring in at least one copy of the gene’s two copies, each inherited from a biological parent. Such mutations leave the gene unable to produce a functional CFTR protein.

Kaftrio, marketed as Trikafta in the U.S., helps the CFTR protein work more effectively through a combination of three CFTR modulators.

Together, these modulators help the protein to fold into its correct 3D shape, keeping it from being quickly degraded and able to stay open longer, allowing more salt to pass in and out of the cell. This prevents mucus from building in organs, affecting their function.

In the EU and the U.K., Kaftrio is given alongside Kalydeco, which further helps to keep the CFTR protein open at the cell surface.

The triple combination was recently approved for CF patients ages 12 and older in the EU with at least one F508del mutation.

These applications are supported by the results of an open-label, global Phase 3 study (NCT03691779) that evaluated Kaftrio’s safety and efficacy in 66 children, ages 6 to 11, over 24 weeks. Participants had either two copies of the F508del mutation, or one copy and one minimal function mutation. They received the full adult dose if their weight was 30 kilograms (about 66 pounds) or more, and half the adult dose if they weighed less.

Results showed that Kaftrio improved lung function and lowered levels of sweat chloride, a sign of CFTR function, in these children. Nutritional status, which is associated with a better lung health and survival, also improved. The medication was generally well-tolerated, with safety data consistent with previous studies in older patients.

“We are committed to working diligently with global regulators to expand the indication for our medicine such that younger people living with CF will also be able to access the triple combination therapy,” Tatsis said.

A similar request to expand Trikafta’s label to children 6–11 is before the U.S. Food and Drug Administration. An agency decision is expected  in June.


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