Trial Profile
A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Status:
Completed
Phase of Trial:
Phase III
Latest Information Update: 12 Apr 2023
Price :
$35
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At a glance
- Drugs Ivacaftor/lumacaftor (Primary)
- Indications Cystic fibrosis
- Focus Adverse reactions; Pharmacokinetics; Registrational
- Sponsors Vertex Pharmaceuticals
- 10 Apr 2023 According to Vertex Pharmaceuticals media release, company today announced based on data from this trial, Health Canada as a partner of Access Consortium, has granted Marketing Authorization for the expanded use of PrORKAMBI (lumacaftor/ivacaftor) for the treatment of cystic fibrosis (CF) in 30 children ages 1 to <2 years who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
- 21 Jan 2019 According to a media release, based on the data of this study, the European Commission has granted approval of the label extension for ORKAMBI (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease.
- 13 Dec 2018 According to Vertex Pharmaceuticals media release, based on the data from this trial, Health Canada has granted Market Authorization for ORKAMBI (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) who have two copies of the F508del CFTR mutation.