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Articles

Safety in Lactation: Mucolytics and other drugs for cystic fibrosis

18 September 2020Additional information relating to breastfeeding To be used in conjunction with individual drug entries for specific information and guidance. There is no evidence to support…
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Medicine Compliance Aid Stability

KalydecoVertex

Vertex
Kalydeco
Tablets f/c 150mg
A1 · Amber 1Stability data is available in an alternative container (not CAs) that may be extrapolated to support storage in CAs.
No special precautions for storage
No special precautions for storage.
29 September 2015

Lactation Safety Information

Very limited published evidence of safety
Small amounts in breast milk probably due to high protein binding
15 September 2019

New Medicines

KalydecoCystic fibrosis in children aged 4 months to 18 years with an R117H CFTR mutation

Information

Kalydeco
Licence extension / variation
Vertex
Vertex

Development and Regulatory status

Launched
Launched
None
November 2020
Nov 20Vertex announces EU approval for ivacaftor to treat eligible infants with cystic fibrosis as early as four months of age. Approval is granted for label extension for ivacaftor granules 25mg to include treatment of infants ages ≥4 months and weighing ≥5 kg with r117h mutation or one of a number of gating (class III) mutations in CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R [4].
Jun 20Approved in EU. Final approval is for Kalydeco 150 mg tablets to extend the use to patients with cystic fibrosis (CF) aged 6 years and older and weighing 25 kg or more who have an R117H mutation in the CFTR gene and for Kalydeco granules 75 mg and 50 mg, to add patients with CF aged 12 months and older and weighing 7 kg to less than 25 kg who have an R117H mutation in the CFTR gene [3].
Apr 20EU positive opinion for a change in the licence for Kalydeco tablets to include treatment of children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation. The new indication will be treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. The CHMP also adopted a change to the indication for Kalydeco granules to include treatment of patients with an R117H CFTR mutation. The new indication for Kalydeco granules will be treatment of infants aged at least 6 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R [1].

Category

Potentiator of the CFTR protein
CF is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia [1]. The UK CF registry 2016 annual report shows 10,461 registered patients, whereby 844 patients are aged <3 years [2].
Cystic fibrosis in children aged 4 months to 18 years with an R117H CFTR mutation
Oral

Evidence based evaluations

22 January 2021EPAR (75mg tab)
3 November 2020EPAR (≥4 months)
9 June 2020EPAR (≥12 months)