New Kaftrio and Kalydeco specialties adapted to the child from 2 to 6 years old

Mucoviscidosis mainly affects the airways and the digestive system.yavdat / iStock / Getty Images Plus / via Getty Images

Dyears The treatment of cystic fibrosis in children, new specialties Kaftrio (ESTEXACAFTOR, IVACAFTOR, TEZACAFTOR) and Kalydeco (IVACAFTOR) are made available in city pharmacy and hospital in addition to the KAFTRIO and KALYDECO ranges [1] :

Kaftrio granules is indicated in association with Ivacaftor (Kalydeco) in the treatment of children with cystic fibrosis from 2 years to less than 6 years old and carrying at least one mutation other than class I of the new kaftrio kalydeco specialties adapted new gene CFTR (cystic fibrosis transmembrane conductance regulator).

Kalydeco granules is indicated :

&#13. However.
• In monotherapy in the treatment of infants at least new kaftrio kalydeco specialties adapted 1 month, young children and children weighing 3 kg to less than 25 kg with cystic fibrosis carrying a mutation R117H gene CFTR or one of the regulation defect mutations (class III) of the gene CFTR next : G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R;
• In association with Ivacaftor/TezacAtor/Elexacaftor Granulated (Kaftrio) in the treatment of children from 2 years old to under 6 years with cystic fibrosis with at least one mutation other than class I of the gene CFTR.

&#13. Meanwhile.

Before being marketed and taken care of in ordinary law, these new Kaftrio and Kalydeco specialties benefited from an early access authorization (AAP) in France.

New kaftrio kalydeco specialties adapted new

New kaftrio kalydeco specialties adapted

Dosages. a galenic shape adapted to the pediatric dosage – New kaftrio kalydeco specialties adapted

The dosage in children from 2 years old to 6 years old is different depending on the child’s body weight.

The dose recommendations are described in new kaftrio kalydeco specialties adapted the following table. extracted from the vidal monograph of pellet kaftrio:

A meal rich in fat before. after taking the medication – New kaftrio kalydeco specialties adapted

An interval of approximately 12 hours must be respected between the morning and evening sockets.

Kaftrio. Kalydeco granules should be administered immediately before. after a meal or a snack rich in fats, such as those containing butter or oil or eggs, cheese, nuts, whole milk or meat.

Remember to avoid consuming foods or drinks containing grapefruit during treatment with Kaftrio new kaftrio kalydeco specialties adapted new or Kalydeco. Consumption of grapefruit can increase exposure to the active ingredients of these drugs.

Instructions for the administration of pellets

Each sachet of Kaftrio and Kalydeco is for single use.

At the time of taking. the contents of the pellet sachet must be mixed with 5 ml of semi-liquid food. liquid new kaftrio kalydeco specialties adapted adapted to the age of the child (fruit compotes, vegetable purees, yogurts, water, milk or fruit juice) and ingested immediately in full.
The food or liquid used for the preparation of the mixture must be at room temperature or lower.
After the mixture, the drug remains stable for one hour and must therefore be ingested within this period.

Patient monitoring

Before initiating the treatment with Kaftrio. Kalydeco, a genotyping must be carried out to confirm the presence of at least one transfer of the gene CFTR new kaftrio kalydeco specialties adapted new Responding to the treatment with the Ivacaftor/TEZACAFTOR/ELEXACAFOR.

A dosage of transaminases (ALAT. ASAT). total bilirubin is recommended in all patients before the establishment of treatment with Ivacaftor, every 3 months during the first year, then at least once a year. The pace of monitoring must be adapted to the patient’s profile. especially in the event of history of liver new kaftrio kalydeco specialties adapted damage or increase in transaminases.

An extension of care for Kalydeco

In parallel with this range extension. Kalydeco 25 mg, 50 mg and 75 mg pellets in a box of 56 bags benefits from an extension of care (reimbursement and approval to communities):

• in the Treatment of infants for at least 1 month to less than 4 months, and children weighing 3 kg to less than 25 kg with cystic fibrosis carrying one of the regulation defect mutations (class III) of the new kaftrio kalydeco specialties adapted new gene CFTR following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

&#13.

In this indication. the Commission for Transparency (CT) of the High Authority for Health (HAS) awarded an important medical service (SMR) to Kalydeco only in patients with one of the 9 regulation defect mutations (class III) of the gene CFTR following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N new kaftrio kalydeco specialties adapted or S549R [2]which excludes patients with a R117H mutation of the gene CFTR.

The IVACAFTOR dosage recommended in the 1 month old infant.. the child weighing by 3 kg to less than 25 kg is described in Table 1 of the Vidal Monograph of Granula Kalydeco.

Administrative identity

List in
Semi -annual initial hospital prescription
Non -limited renewal

Kaftrio 60/40/80 mg pellets. box of 28 sachets, CIP 3400930280348, prix public TTC = 9 130,40 euros [3]
Kaftrio 75/50/100 mg pellets, new kaftrio kalydeco specialties adapted new box of 28 sachets, CIP 3400930280355, prix public TTC = 9 130,40 euros [3]

Kalydeco 59.5 mg pellets. box of 28 sachets. CIP 3400930280768 [4]
Kalydeco 75 Mg Granulés :

• Box of 28 CIP bags 3400930280751, prix public TTC = 5 151,65 euros [4]
• Box of 56 sachets, CIP 3400930041000, prix public TTC = 10 170,42 euros [4]

Refundable at 65 % on exceptional drug prescription (cf. Framed 2 and 3) new kaftrio kalydeco specialties adapted [5 et 6 – Fiche d’information thérapeutique]
Approval for communities (cf. Framed 2. 3) [7 et 8]
Green Laboratory Pharmaceuticals France