3 CFTR modulators guaranteed public reimbursement in England
Vertex treatments Kaftrio, Symkevi, Orkambi to be covered
Vertex Pharmaceuticals and England’s National Health Service (NHS) have reached an agreement that allows all current and future eligible cystic fibrosis (CF) patients to access three of the company’s approved CFTR modulators at low or no out-of-pocket cost.
Kaftrio (elexacaftor/tezacaftor/ivacaftor, sold as Trikafta in the U.S.), Symkevi (tezacaftor/ivacaftor, sold as Symdeko in the U.S.) and Orkambi (ivacaftor/lumacaftor) are covered by the agreement, according to a company press release.
The agreement, which also guarantees access to any future license extensions of the medicines, follows a positive recommendation by the National Institute for Health and Care Excellence (NICE).
Kaftrio/Trikafta, in combination with Kalydeco (ivacaftor), will be available at low or no cost to CF patients 2 years and older who carry have at least one F508del mutation — the most common CF-causing mutation — in the CFTR gene. Patients 6 and older who have either two F508del mutations or one F508del mutation and another eligible CFTR mutation are eligible for reimbursement of treatment with Symkevi/Symdeko, in combination with Kalydeco. Orkambi will be available for patients 1 and older carrying two F508del mutations.
Wales, Northern Ireland to follow
NICE’s recommendations are also based on undisclosed discount prices for each therapy that were determined in commercial agreements between Vertex and NHS England.
According to U.K.’s Cystic Fibrosis Trust, Wales and Northern Ireland will also follow NICE’s recommendation, while talks between Vertex and NHS in Scotland for a commercial deal were continuing.
Vertex, NHS England, and NICE also committed to work towards rapid access for all eligible patients to the company’s new CFTR modulator, vanzacaftor/tezacaftor/deutivacaftor, should it be approved by the U.K. Medicines and Healthcare products Regulatory Agency, the company said.
Formerly known as VX-121/tezacaftor/VX-561, the experimental triple-combination therapy contains two more recently developed investigational CFTR modulators in addition to tezacaftor, one of the modulators used in Kaftrio/Trikafta.
In CF, CFTR mutations lead to a faulty CFTR protein or no protein at all being produced. As CFTR normally controls the flow of water through cells that produce mucus, sweat, saliva, tears, and digestive enzymes, these mutations lead to the excessive production of mucus in several organs and salty sweat.
CFTR modulators are a class of therapies that target the underlying cause of CF and can increase the functionality of the defective CFTR protein in people with specific disease-causing mutations. This allows more normal mucus production across several organs, which ultimately eases CF symptoms and slows the progression of damage to the body’s organs.
Genetic testing is usually conducted as part of the CF diagnosis process to determine what underlying mutations patients have and the modulator(s) for which they are eligible.
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