PBAC's latest decision on Lumacaftor with ivacaftor: Recommended with restriction (2023). Considered for Treatment of cystic fibrosis in patients aged 1 to less than 2 years who are homozygous for the F508del mutation in the CFTR gene.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
—
ICER not stated
Submissions
5
first 2016
Submissions
5
2016 → 2023
Eligible population
Children aged 1 to less than 2 years with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
Single-arm
Primary endpoint
Safety
Pivotal trial size
60 patients
Key trials
Study 809-122 (NCT03601637)
Comparator
best supportive care
ICER note
ICER value not stated in the provided PSD text; text appears to be truncated at the economic evaluation section.
Submission history
Mar 2016: Recommended with restriction · Authority Required
Nov 2016: Not recommended · Authority Required
Jul 2018: Deferred · Authority Required
Jul 2018: Recommended with restriction · Authority Required
Jul 2023: Recommended with restriction · Authority Required