NHS, Vertex Strike Deal to Make Kaftrio Available in England

NHS, Vertex Strike Deal to Make Kaftrio Available in England
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NHS England and Vertex Pharmaceuticals have reached an agreement that will allow access to Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor), a triple combination therapy for cystic fibrosis (CF) to be used in a combination regimen with Kalydeco, as soon as the medication is approved by the European Commission.

The agreement gives reimbursed access to Kaftrio (marketed as Trikafta in the U.S.), if approved in Europe, expanding the previous reimbursement agreement for Vertex’s other approved CF medications, namely Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor combo). The agreement also covers future indications for these medications.

“I’m pleased that NHS England has recognized the value of Kaftrio, and that Vertex and NHS England have been able to work quickly, collaboratively and flexibly to expand the existing reimbursement agreement to include the triple combination therapy in advance of the medicine being licensed,” Reshma Kewalramani, MD, president and CEO of Vertex, said in a press release.

“This will ensure that eligible patients in England will be among the first in Europe to benefit from access to this innovative medicine upon approval,” Kewalramani said.

The Committee for Medicinal Products for Human Use (CHMP), part of the  European Medicines Agency, recently issued a positive opinion on the use of Kaftrio, in combination with Kalydeco, as a treatment for CF in people 12 and older who have either two F508del mutations (the most common CF-causing mutation), or one F508del mutation and one minimal function mutation in the CFTR gene (the gene defective in CF).

This opinion was based on results from the AURORA clinical trials (NCT03525444 and NCT03525548), which demonstrated that Kaftrio significantly reduced exacerbations and improved lung function in people with CF who have one or two F508del mutations.

The new agreement covers access to Kaftrio for people with CF in England who have two F508del mutations, or one F508del mutation and one minimal function mutation.

“It is fantastic news that a deal has been done between NHS England and Vertex Pharmaceuticals and Kaftrio will now be available to thousands of people across England in the coming weeks. This will truly save lives,” David Ramsden, chief executive of the U.K. Cystic Fibrosis Trust, said in a press release.

In addition, the agreement allows Kaftrio and other Vertex medications to be prescribed for individuals with rare mutations that may be covered by licensing decisions by the U.S. Food and Drug Administration (FDA).

“We have also agreed a flexibility so that clinicians will be able to use that not only within its European license and any other future indications but also other drugs in the portfolio for rare mutations that might be covered by licensing by the US FDA as well,” Simon Stevens, NHS chief executive, said in a Health and Social Care Select Committee meeting, according to the Trust’s press release.

“This is a very significant day for cystic fibrosis patients that really tackles the underlying cause of the disease by helping lungs work effectively,” Stevens said.

The agreement also may pave the way for similar agreements in Scotland, Northern Ireland, and Wales.

Alexandra Andrews, a 45-year-old CF patient from Nottingham, England, who has been taking Kaftrio since May 2020 on compassionate grounds, said: “It’s been amazing. It’s improved my quality oaf life no end. I can actually do more of the little things in life. I can exercise again and I feel healthier. This drug has made a massive difference and it’s helped me to feel better mentally too. I can now have a conversation without feeling out of breath, without struggling to breath. It’s like a miracle drug and it’s given me a new lease of life.”

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.
Total Posts: 336

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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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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