MHRA Approves Vertex’s Kaftrio combination with ivacaftor for Children With cystic fibrosis
pharmafile | January 12, 2022 | News story | Research and Development |
Vertex Pharmaceuticals have announced the MHRA approval of the label extension of Kaftrio® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of cystic fibrosis (CF) in patients in England, Scotland and Wales.
The treatment has been approved for children ages 6 to 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Access to Kaftrio® in a combination regimen with ivacaftor for these patients is included in the long-term agreements in place between Vertex and the NHS in England, Scotland and Wales.
Over 10,830 people in the UK have CF. CF is an inherited, debilitating, life-shortening condition, that causes progressive damage to organs across the body from birth. Symptoms usually begin in early childhood and grow worse over time, with lungs and digestive system becoming increasingly damaged. There is currently no cure for CF, and half of people in the UK with CF die before they are 36.
The daily impact of treatment for CF is significant, and it can take up to four or more hours involving, nebulisers, physiotherapy and up to 70 tablets a day. Symptoms include a difficulty putting on weight and growing, a yellowing of the skin and whites of the eyes, wheezing, coughing, shortness of breath and damage to the airways, recurring chest infections, and bowel obstruction in newborn babies (meconium ileus), for which surgery may be needed.
Jane Davies, Professor of Paediatric Respirology & Experimental Medicine at the National Heart and Lung Institute, Imperial College London and Honorary Consultant in Paediatric Respiratory Medicine at Royal Brompton & Harefield Hospital, Guy’s & St Thomas’ NHS Trust, London, commented: “Favourable safety and efficacy data have supported this license extension, from which the majority of children with CF aged 6 years and older will be able to benefit. On behalf of the clinical teams, I wish to thank all the children and families involved in this trial for their extremely valuable contributions.”
Ana Ovey
