NICE recommends innovative cystic fibrosis therapy
Recommendation of the once-daily triple therapy increases the number of eligible cystic fibrosis patients who can access treatments in the UK.
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Recommendation of the once-daily triple therapy increases the number of eligible cystic fibrosis patients who can access treatments in the UK.
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of four new medicines plus the extension of therapeutic indications for 16 medicines.
Following a milestone approval by the European Commission in July 2023, Vertex Pharmaceuticals has been granted a label expansion for its cystic fibrosis medicine KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) with ivacaftor.
Children with cystic fibrosis aged six to11 years could benefit from Kaftrio® in combination with ivacaftor, as a treatment targeting the underlying cause of the disease.
In its March meeting the EMA’s human medicines committee recommended five medicines for approval and concluded its review of regdanvimab as a treatment for COVID-19.
The European Commission and European Medicines Agency have approved the triple combination Kaftrio for use in certain cystic fibrosis patients, triggering the drug to be accessible on the UK’s National Health Service.