Kaftrio Approved for Children Ages 6 and Up in European Union, UK

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by Marisa Wexler, MS |

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The European Commission has expanded its approval of Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to treat children with cystic fibrosis (CF) as young as 6 who have the most common type of CF-causing mutation.

The Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. approved a similar expansion.

“Today both the European Commission and MHRA have agreed to license Kaftrio for use in children aged 6–11 years old. More than 1,500 children across the UK and Northern Ireland stand to benefit from this drug and limit the damage CF does in these critical early years,” David Ramsden, chief executive at the Cystic Fibrosis Trust, said in a press release.

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“We are delighted that KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is now approved for these young patients in the European Union. It provides a new treatment option for physicians to help treat the underlying cause of this devastating disease early in life,” Reshma Kewalramani, MD, said in a separate release. Kewalramani is president and CEO of Vertex Pharmaceuticals, which markets Kaftrio and Kalydeco.

CF is caused by genetic mutations that affect the production of the protein CFTR. Kaftrio is an oral therapy containing three CFTR modulators, which are medications that can bind to the protein and increase the functionality of people with specific mutations.

In Europe, Kaftrio is approved for patients with at least one copy of the F508del mutation — the most common CF-causing mutation, present in about 90% of patients. The European Commission had approved the medication for patients ages 12 and up last year, based on clinical trial data showing it could improve lung function.

Kaftrio with Kalydeco “has shown clinical benefit since its availability last year for people with CF ages 12 and above,” said Marcus A. Mall, MD, of Charité University Medical Center Berlin.

Vertex’s application to expand the approval to children as young as 6 was supported by clinical trial data that showed the therapy’s safety profile in these young children is similar to what is seen in older patients.

“CF is a progressive disease, in which symptoms and organ damage manifest very early in life. As a physician, I welcome the approval of this medicine for this younger age group, as it will help us treat eligible children with CF as early as 6 years old,” Mall said.

“This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF,” Kewalramani said.

Kaftrio is expected to be available soon after approval in Austria, Northern Ireland, and Denmark, where long-term reimbursement agreements are already in place. Availability also is expected soon in Germany and other countries with provisions for access. Vertex is working with other authorities across Europe on reimbursement agreements to further expand access.

Kaftrio’s availability has been welcomed by the CF community. Catherine Farrer, from London, whose 7-year-old daughter Kate has CF, said she’s been waiting her daughter’s whole life for this moment.

“As we sit in hospital having IVs for the third time in 13 months, it is impossible to put into words what this means for not just Kate, but for our whole family, extended family, friends, medical team and everyone who plays a part in trying to keep Kate well and living a ‘normal’ life,” Farrer said.

“While this is a good day, Kaftrio is not a cure, and the treatment sadly will not work for every child,” Ramsden said. “The Cystic Fibrosis Trust won’t stop until everyone with CF has access to the support and treatments they need.”

Kaftrio, marketed as Trikafta, is also approved for patients ages 6 and up in New Zealand, Switzerland, and the U.S., and it is up for a similar expansion in Canada.