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Vertex’s triple combination CF therapy to become available on the NHS

The drug will immediately become available to thousands of cystic fibrosis patients

NHS England has reached an agreement with Vertex for immediate access to the pharma company’s transformative triple combination therapy for cystic fibrosis (CF).

The landmark deal for the combination therapy, Kaftrio (ivacaftor/tezacaftor/elexacaftor), was made possible thanks to previous negotiations between Vertex, NHS England and the UK’s National Institute for Health and Care Excellence (NICE).

Last October, the three parties finally reached an agreement for access to Vertex’s other cystic fibrosis drugs Orkambi (lumacaftor/ivacaftor), Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor).

Under the terms of the deal, NHS access to the pharma company’s triple therapy would be ensured as soon as it received approval from the European Medicines Agency (EMA). Vertex scored EMA authorisation on Friday, allowing for immediate access to the drug on the NHS for thousands of CF patients in England.

Through the commercial deal, NHS England has also secured equivalent terms for CF patients in Wales, Northern Ireland and Scotland. According to NHS England, the deal will span four years to allow for further data on the medication to be collected.

The terms of the deal also mean that up to 300 CF patients with rare genetic mutations, who previously were not included within the scope of the EMA’s licensing considerations, will now be able to access Kalydeco and Symkevi.

Although the European licence for Kaftrio, named Trikafta in the US, only covers patients aged 12 years and older, younger children and those over 12 years of age who are not eligible will be able to benefit from access to Vertex’s other CF drugs. If the licence for Kaftrio is extended in the future, access to the triple therapy will become immediately available to these groups, said the NHS in a statement.

“The licensing of Kaftrio today marks a step change in the treatment of cystic fibrosis. With agreements between Vertex and governments across the UK already in place, thousands of eligible people across the UK can now discuss with their clinical team how they can start this treatment as soon as possible,” said David Ramsden, chief executive of the Cystic Fibrosis Trust.

“It’s also great news that more mutations have been added to those eligible for Kalydeco and Symkevi, giving more children and adults with CF a disease modifying treatment option,” he added.

In clinical trials, Kaftrio significantly improved lung function in CF patients with two copies of the F508del mutation or one copy of the F508del mutation with a minimal function mutation.

Article by
Lucy Parsons

24th August 2020

From: Regulatory

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