A treatment against cystic fibrosis combining two drugs can now be administered to certain children aged 6 to 11 years with this pathology. Until now, only those over the age of 12 were allowed to take it.
- 7,300 people are affected by cystic fibrosis in France.
- 108 children with cystic fibrosis were born in France in 2020.
It is now official: a treatment against cystic fibrosis combining two drugs – Kaftrio and Kalydeco – is now extended to certain children aged 6 to 11 suffering from this pathology. The Haute Autorité de Santé (HAS) announced this Tuesday, March 29 in a communicated. Until now, only children over the age of 12 were allowed to take it.
A genetic disease
Cystic fibrosis is an inherited genetic disease that is usually diagnosed at birth or in the first months of life. The main organs affected are the lungs, the pancreas, the sweat glands (located under the skin and whose role is to produce sweat) and, in men, the sex glands. It is in particular the alteration of the CFTR gene that causes respiratory complications.
288 children affected by the early access authorization
The health authority nevertheless specifies that this authorization for early access to treatment will only concern children aged 6 to 11 years “who are heterozygous for the F508del mutation of the gene CFTR and carriers of a minimal function CFTR gene mutation”. In other words, only for certain small patients with cystic fibrosis. According to the HAS, its decision could concern 288 children of this age group suffering from this disease.
Why this category only?
“Until now, the treatments offered to children aged 6 to 11 heterozygous for the F508del mutation of the CFTR gene and carriers of a mutation of this same gene with minimal function were only symptomatic and not specific for the type of mutation from which they suffer.”, specifies the HAS, which recognizes “the presumed innovative nature of this combination of drugs which, in the long run, would allow patients to breathe better, to be sick less often and to regain weight”.
Reimburse this treatment
But what does early access authorization mean? While waiting for the marketing authorization to be validated, HAS can grant early access authorizations which allow the treatment to be taken and reimbursed by Social Security. This will now be the case for 6 to 11 year olds heterozygous for the F508del mutation of the CFTR gene and carriers of a mutation of this same gene with minimal function.
Marketing Authorization granted in August 2020 for ages 12 and over
The marketing authorization (AMM) for this treatment for patients over 12 years of age with cystic fibrosis – and carriers of at least one F508del mutation of the CFTR gene – was validated in August 2020. Since January 7 , an extension of Marketing Authorization was granted for four categories of patients aged 6 to 11 suffering from this disease: homozygous for the F508del mutation of the gene CFTRheterozygous for the F508del mutation of the gene CFTR and carriers of a gene mutation CFTR with residual function, heterozygous for the F508del mutation of the gene CFTR and carriers of a “gating” mutation of the gene CFTRheterozygous for the F508del mutation of the gene CFTR and carriers of a gene mutation CFTR at minimum function.
Do not delay treatment
“In order not to delay access to this treatment, and pending its coverage under common law, HAS authorizes early access to this combination of treatments for this last category of children aged 6 to 11 years (heterozygotes for the F508del mutation of the CFTR gene and carriers of a minimal function CFTR gene mutation)”, concludes the HAS in its press release.
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