England's NICE Backs Vertex’s CF Drugs Kaftrio, Symkevi & Orkambi After Initial Rejection
Executive Summary
Cystic fibrosis patients in England will be able to access Vertex’s three combination therapies after the company announced an extended reimbursement agreement with the National Health Service and a positive opinion from the country’s HTA body, NICE.
Vertex Pharmaceuticals and NHS England have extended an existing long-term reimbursement agreement for the cystic fibrosis drugs Kaftrio (ivacaftor/tezacaftor/elexacaftor), Symkevi (tezacaftor/ivacaftor) and Orkambi (lumacaftor/ivacaftor), the US-based company announced on 20 June.
The agreement includes access to any further indication extensions for the medicines, as well as the existing indications for which the drugs are authorized, Vertex said.
The announcement was made concurrently with a decision from NICE, the health technology assessment (HTA) body for England and Wales, to recommend reimbursement of the medicines for patients with CF who meet certain criteria. (Also see "NHS England And Vertex Strike “Lightening Quick” CF Kaftrio/Trikafta Deal" - Pink Sheet, 2 Jul, 2020.).
In its latest draft guidance, NICE said that all three drugs – which are cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies – were now within the acceptable cost-effectiveness ranges and therefore should be reimbursed on the NHS in England for eligible patients with CF. NICE originally rejected the three medicines in November 2023.
The final appraisal document details eligibility criteria for each medicine, which for Kaftrio, the newest of the three combinations, is in patients with CF who are aged six years or older and have at least one F508del mutation in the CFTR gene. This is the same indication for which the drug is authorized in the UK and EU.
The road to long-term reimbursement for Vertex’s various combination therapies for CF in England has been uncertain at times, despite the NHS describing Kaftrio as a “miracle” treatment in 2022, when the drug’s indication was extended to include younger children with the disease. (Also see "At Last: Vertex Strikes CF Deal In England" - Pink Sheet, 24 Oct, 2019.).
The original agreement between NHS England and NICE for access to Symkevi and Orkambi began in 2019, and Kaftrio was added in 2020. The drugs were meant to be available on the NHS while further evidence was generated to support a long-term reimbursement decision.
In November 2023, NICE released draft guidance in which it turned down Kaftrio, Symkevi and Orkambi for NHS use in CF, saying the medicines were too expensive to be used within the NHS and did not meet the HTA body’s cost-effectiveness criteria.
Despite the positive reimbursement decision from NICE, some patients with CF have rare gene mutations that mean they would not be eligible for treatment with Kaftrio, Symkevi or Orkambi under the current guidance. The Cystic Fibrosis Trust, a charity, said that NHS England has stated that it wants to “explore ways for anyone who might benefit from modulators to have access, if their CF care team recommends this.”
Vertex said it was also working with NHS authorities in Scotland, Wales and Northern Ireland to finalize “similar access agreements” as soon as possible. The company added that its CF medicines were “broadly available in over 60 countries worldwide,” including Australia, France, Italy, Germany, Ireland, the Netherlands, Spain and the US.
Working On Access To Vanzacaftor Triple
According to Vertex, NHS England and NICE had also “committed to work together” to ensure “rapid access” to its next-in-class CF combination candidate, which contains the active ingredients vanzacaftor, tezacaftor and deutivacaftor, once it has been approved by the MHRA. The candidate, dubbed vanzacaftor triple, was filed for EU approval this month. (Also see "First Drug Treatment For Myopia Among 14 New EU Filings" - Pink Sheet, 17 Jun, 2024.).
A marketing authorization application for vanzacaftor triple has also been filed with the US Food and Drug Administration, and the company said in May that it planned to complete regulatory submissions for the new product in Great Britain, Canada, Switzerland, Australia and New Zealand later this year.
The European Medicines Agency first authorized Kaftrio in 2020, 10 months after it was authorized in the US. (Also see "Vertex's Triple CF Combo Gets EU Approval Nod; Rejection For Daiichi Sankyo" - Pink Sheet, 26 Jun, 2020.).
Orkambi was first authorized in the EU in 2015 and Symkevi in 2018. Vertex and NHS England also underwent years of negotiations around the reimbursement of these two drugs in England. (Also see "Vertex Commits To Novel Reimbursement Plan For Orkambi In UK, Despite Setback " - Pink Sheet, 22 Mar, 2018.).