Northern Ireland Reaches Deal With Vertex for Access to Kaftrio

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by Forest Ray PhD |

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Kaftrio, Northern Ireland

The government of  Northern Ireland reached a deal with Vertex Pharmaceuticals to make Kaftrio (ivacaftor/tezacaftor/elexacaftor) available for cystic fibrosis (CF) patients, once the medication receives its European license.

The agreement follows similar ones that the company made recently with England and Wales.

“We’re delighted to see Northern Ireland and Vertex Pharmaceuticals reach a deal on Kaftrio, ensuring that hundreds of people across Northern Ireland will soon get access to this truly life-saving medicine,” Keith Brownlee, director of policy, programs, and support at the Cystic Fibrosis Trust, said in a press release.

Kaftrio is a triple combination therapy, containing 100 mg of elexacaftor, 50 mg of tezacaftor, and 75 mg of ivacaftor. It is meant to be taken with Kalydeco (150 mg), also marketed by Vertex. A similar triple combination therapy named Trikafta has been approved in the U.S.

Under the agreement, patients ages 12 and older and who have either two copies of the F508del mutation, or one copy of F508del mutation and one copy of a minimal function mutation in the CFTR gene (the gene defective in CF) will be eligible for Kaftrio treatment.

The F508del mutation is the most common CF-causing mutation.

According to the Cystic Fibrosis Trust, this agreement marks a big moment for the CF community. The Trust, along with CF patients and advocates, have long campaigned for greater access to life-saving therapies.

Writing in a blog on the CF Trust’s website, Brownlee stated in late June that “Kaftrio will likely receive its marketing authorisation in late summer or early autumn” from the European Medicines Agency (EMA).

In June, the EMA’s Committee for Medicinal Products for Human Use issued a positive opinion on the use of Kaftrio, in combination with Kalydeco, as a treatment for CF. This positive opinion was based on results from the AURORA clinical trials (NCT03525444 and NCT03525548), which showed that Kaftrio significantly improved lung function and reduced disease exacerbations in CF patients.

“While this is a special moment, there is more to do, and we will not stop until everyone with cystic fibrosis across the UK has access to life saving drugs,” Brownlee said.

Liam McHugh, 58, from County Tyrone in Northern Ireland, has a daughter who was born with cystic fibrosis more than 28 years ago.

“Ever since I’ve dedicated my life to fundraising and campaigning for life-saving drugs for people with the condition,” he said. “So news that Kaftrio will be available to people in Northern Ireland is incredible and is going to extend lives.”