2024-02-13 16:57:55
Present in approximately 70% of patients, the Delta F508 mutation in the CFTR gene is the most common.
In the treatment of patients over 6 years old with cystic fibrosis and non-carriers of the F508del mutation (off-label situation as of February 13, 2024 – cf. Boxed), the specialties KAFTRIO and KALYDECO in tablet form have, since February 5, 2024, been made available in pharmacies for indoor use (PUI) according to two devices [1] :
- via and early access device (AP), including for patients previously treated in a compassionate setting. Early Access Authorization (AAP) was granted on September 7, 2023 [2] in the following indication, subject to compliance with the therapeutic use protocol – PUT :
- patients with an ivacaftor/tezacaftor/elexacaftor response mutation on the database in vitro available;
- via the compassionate prescribing (CPC) framework. This system has been open since June 1, 2023. It now applies to patients not eligible for AP (except those who present two mutated genes predictive of the absence of CFTR protein synthesis).
Box – Specialties affected by the new conditions of provision
- KAFTRIO 37.5 mg/25 mg/50 mg and KAFTRIO 75 mg/50 mg/100 mg film-coated tablet (ivacaftor + tezacaftor + élexacaftor): according to the current marketing authorization (AMM), these specialties are indicated in combination with ivacaftor in the treatment of patients with cystic fibrosis aged 6 years and older with at least one F508del mutation in the gene CFTR (cystic fibrosis transmembrane conductance regulator) ;
- KALYDECO 75 mg and KALYDECO 150 mg film-coated tablet (ivacaftor): according to the Marketing Authorization, these specialties are indicated:
- as monotherapy in the treatment of adults, adolescents and children aged 6 years and over, and weighing 25 kg and over, with cystic fibrosis carrying an R117H mutation of the gene CFTR or one of the regulatory defect mutations (class III) of the gene CFTR (cystic fibrosis transmembrane conductance regulator) following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R;
- in combination with tezacaftor/ivacaftor tablets, for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis who are homozygous for the F508del mutation or heterozygous for the F508del mutation and carry one of the following mutations in embarrassed CFTR : P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G et 3849+10kbC→T ;
- in combination with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis who have at least one F508del mutation in the gene CFTR.
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