Scottish Government and Vertex Strike Deal for Access to Kaftrio – Cystic Fibrosis News Today

The Scottish Government and Vertex Pharmaceuticals have reached a deal to make Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) available for people with cystic fibrosis (CF) once the medication receives its European license.

This follows similar agreements in the three other U.K. countries, EnglandWales, and Northern Ireland. According to the U.K. Cystic Fibrosis Trust, through these four agreements an estimated 90% of people with CF in the U.K. will have access to Kaftrio after its approval in Europe.

“It’s fantastic that the Scottish Government and Vertex Pharmaceuticals have agreed a deal on Kaftrio. Available to people across Scotland in the coming weeks, this new drug will truly save lives,” David Ramsden, chief executive of the U.K. Cystic Fibrosis Trust, said in a press release.

Kaftrio (marketed as Trikafta in the U.S.) is a triple combination therapy containing three CFTR modulators — medications that help the protein CFTR, which is defective in CF, work more effectively. The triple therapy is meant to be taken with Kalydeco, which also is marketed by Vertex.

The new agreement gives access to Kaftrio to people with CF in Scotland who are 12 or older and have either two copies of the F508del mutation, or one copy of F508del and one copy of a minimal function mutation in the CFTR gene. F508del is the most common CF-causing mutation.

The agreement also covers individuals with certain rare mutations who are covered by the licensing decisions of the U.S. Food and Drug Administration (FDA), which approved the treatment in 2019.

The Committee for Medicinal Products for Human Use, a part of the European Medicines Agency (EMA), in June issued a positive opinion on the use of Kaftrio as a treatment for CF. The positive opinion was based on results from the AURORA clinical trials (NCT03525444 and NCT03525548), which demonstrated the treatment significantly improved lung function and reduced exacerbations in people with CF.

A final decision from the EMA is expected in late summer or early autumn.

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

Shona Young, a 25-year-old with CF from Glasgow, Scotland, said: “Because of Kaftrio, my whole life is going to change. I will, for the first time, be able to plan ahead for a longer future. I’m overwhelmed and am so excited about the new possibilities this treatment will open up for me and for so many others with cystic fibrosis,” she said. “Kaftrio is what we have all been waiting for and I couldn’t be more grateful that it exists.”

Marisa, a science writer, holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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