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The Respiralia Group joins the Spanish Federation of Cystic Fibrosis in the claim of the children’s Kaftrio© on the National CF Day.
National Cystic Fibrosis Day
On the occasion of World Cystic Fibrosis Day, which is commemorated on September 8, the Respiralia Group, formed by the Cystic Fibrosis Balearic Association and the Respiralia Foundation, join the Spanish Cystic Fibrosis Foundation (FuEFQ), the Spanish Federation of Cystic Fibrosis (FEFQ) and the Spanish Society of CF (SEFQ) to urge the laboratory Vertex Pharmaceuticals and the Ministry of Health to reach a funding agreement at the next meeting of the CIPM in late September, to include the pediatric indication of Kaftrio© in the National Health System (SNS) and not further lengthen the wait for 500 children with Cystic Fibrosis in Spain who continue to suffer an irreversible deterioration of their health.
The revolution of CFTR protein modulators
Cystic Fibrosis (CF) has undergone a revolution in recent years in terms of treatment. The development of CFTR protein modulators, and their extension to more and more mutations and age groups, is leading to a considerable improvement in the quality of life of many people with CF. These treatments do not cure, but they slow down the deterioration caused by the disease.
Kaftrio© marks a before and after in Cystic Fibrosis
The last of these modulators, Kaftrio©, was included with a delay compared to surrounding countries in our National Health System (NHS), on December 1, 2021, for people with CF over 12 years of age with at least one copy of the F508del mutation.
This comprises approximately 70% of the CF population in Spain. Almost simultaneously, the European Medicines Agency (EMA) extended the indication of this treatment to girls and boys between 6 and 11 years of age, and it was authorized in January by the European Commission for marketing in Europe.
History is about to be repeated
What happened with the adult indication is about to be repeated and, eight months later, 500 children with Cystic Fibrosis in Spain are still waiting to have access to this revolutionary treatment.
Last May, at the meeting of the Interministerial Commission on Drug Prices (CIPM), it was agreed to finance the pediatric indications of two of Vertex’s CFTR modulators (Kalydeco© and Orkambi©). However, the other two (Kaftrio© and Symkevi©) were not approved and, as of today, negotiations continue between the laboratory and the Ministry of Health to obtain authorization for these pediatric indications at the next CIPM to be held at the end of September.
Testimonials from family members of children with Cystic Fibrosis