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Vertex Pharmaceuticals has announced that the European Commission has granted approval to extend the indication of the drug Kaftrio (ivacaftor / tezacaftor / elexacaftor) in combination with ivacaftor for the treatment of children with Cystic Fibrosis aged 2-5 years with at least one copy of the F508del mutation in the CFTR gene, regardless of their other mutation.
Next steps
With this marketing authorisation, the laboratory can now apply for this new indication for Kaftrio to be included in the national health system of each EU country.
We hope that, in Spain, Vertex will apply for marketing so that the Ministry of Health can initiate the funding procedure and that it can be approved by the Interministerial Commission on Medicines Prices (CIPM) as soon as possible.
Dr. Óscar Asensio, President of the Cystic Fibrosis Spanish Foundation
“Once again, we welcome an extension of the indication for a highly effective drug for a severe and degenerative minority disease to younger children. After months of waiting and revisions following FDA approval, Europe approves Kaftrio for children aged 2-5 years – another step towards CF treatment with diagnosis of the disease following neonatal screening. We need to move forward to prioritise fast-track approvals for these situations for drugs that are fully tested in their age extensions. Children with Cystic Fibrosis are doubly discriminated against in access to innovative drugs. We should avoid delaying the arrival of the drug to Cystic Fibrosis patients once there is sufficient evidence of its efficacy in degenerative diseases. Access should not be linked to the negotiation of funding or the bureaucracies of each country after approval by the European Medicines Agency, in which the member states are already involved”.
Teresa Llull, President of the Respiralia Foundation
“Una vez más, acogemos con satisfacción la ampliación a niños más pequeños de la indicación de un medicamento muy eficaz para una enfermedad minoritaria grave y degenerativa. Tras meses de espera y revisiones tras la aprobación de la FDA, Europa aprueba Kaftrio para niños de 2 a 5 años, un paso más hacia el tratamiento de la FQ con diagnóstico de la enfermedad tras el cribado neonatal. Tenemos que avanzar para dar prioridad a las aprobaciones por la vía rápida en estas situaciones para medicamentos que estén totalmente probados en sus extensiones de edad. Los niños con Fibrosis Quística están doblemente discriminados en el acceso a fármacos innovadores. Deberíamos evitar retrasar la llegada del fármaco a los pacientes con Fibrosis Quística una vez que hay suficiente evidencia de su eficacia en enfermedades degenerativas. El acceso no debe estar ligado a la negociación de la financiación ni a las burocracias de cada país tras la aprobación por la Agencia Europea del Medicamento, en la que ya están implicados los estados miembros”.
History
Currently in Spain, the drug Kaftrio in combination with ivacaftor is available for the treatment of people with Cystic Fibrosis from 6 years of age from 1 November 2022..
