Vertex Finally Reaches Pricing Deal With England’s NHS for Cystic Fibrosis Drugs

2024-06-21

临床3期上市批准

Pictured: Vertex Pharmaceuticals' headquarters in Boston, Massachusetts/iStock, hapabapa Vertex Pharmaceuticals on Thursday announced that it has entered into an extended, long-term reimbursement agreement with England’s National Health Service, granting access to its cystic fibrosis treatments Kaftrio (ivacaftor/tezacaftor/elexacaftor), Symkevi (ezacaftor/ivacaftor) and Orkambi (lumacaftor/ivacaftor). The biotech did not disclose specific details of the deal, only revealing that it would also apply to future license extensions of these drugs. Vertex is working on similar deals in Scotland, Wales and Northern Ireland. Ludovic Fenaux, senior vice president of Vertex International, said in a statement that the company is “delighted” to have reached a deal with the National Health Service (NHS), adding that the reimbursement agreement will ensure “long-term access” to Kaftrio, Symkevi and Orkambi for cystic fibrosis (CF) patients in England. Vertex is currently in talks with NHS England as well as the drug pricing watchdog National Institute for Health and Care Excellence (NICE) to ensure rapid access to the biotech’s next-generation triple combination CF treatment. In a Phase III readout posted in February 2024, the investigational therapy, dubbed vanza triple, matched Kaftrio in terms of improving patients’ lung function, and outperformed the approved treatment in terms of sweat chloride levels. Thursday’s reimbursement deal comes after the NICE in October 2023 declined to recommend Kaftrio, Symkevi and Orkambi for use in CF patients. At the time, the watchdog said that while the drugs’ efficacy data support their clinical benefit, they were too expensive to be covered. “The most likely cost-effectiveness estimates for [Kaftrio, Orkambi and Symkevi] are above the range that NICE considers an acceptable use of NHS resources,” the agency wrote. However, earlier this week, NICE reversed its recommendation. In its final draft guidance, the watchdog said that given the severity of CF and its effects of quality and length of life, the cost-effectiveness estimates for Kaftrio, Symkevi and Orkambi now fell within its threshold of acceptable use of NHS resources. While NICE’s recommendation and the NHS deal names three of Vertex’s CF drugs, the centerpiece is Kaftrio, which the watchdog said yielded better short- and long-term improvements than Symkevi and Orkambi. Kaftrio is also known as Trikafta in the U.S. and is a three-drug combo that first won the FDA’s approval in October 2019, allowing its use in patients aged 12 years and older bearing at least one copy of the F508del mutation in the CTFR gene. Trikafta works by increasing the number and improving the function of F508del-CFTR proteins at the cell surface. Trikafta won additional approvals in 2021, for use in CF patients ages 6 to 11 years, and in 2023, for patients 2 through 5 years. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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