Skip to main content

ivacaftor

Generic: IVACAFTOR·Brand: KALYDECO, Kalydeco

ivacaftor: sourced relationships with 1 company · 1 clinical trial tracked · 2 FDA decisions.

ivacaftor evidence translation

What we can confirm

2 FDA records · 1 tracked trial

What it means

Source-backed records connect ivacaftor to VRTX; that link alone does not prove the drug works.

What to watch

Watch active trials and any change in FDA or trial status.
BioSniper AIPage evidence connected

Prepared research question

How could ivacaftor → VRTX affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence1 indications · 0 study sponsors

Related indications

Cystic Fibrosis

Study sponsors

No additional listed-company sponsor is linked.

Dated Pipeline Records (1)
CompanyDated record stage
VERTEX PHARMACEUTICALS INC / MA (VRTX)
Reported 2012-01-31 · Current development: unknownSource
Approved
Clinical Trial Registry Records (1)

A Phase 2, Randomized, Multicenter, Double Blind, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 in Combination With Ivacaftor for 12 Weeks in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation With an Open-Label Extension

NCT02070744Registry updated 2025-09-24·Phase 2
Completed

Primary endpoint: PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

InterventionalRandomizedtriple40 enrolledStarted 2014-03Completion 2016-0523 sites · United StatesVertex Pharmaceuticals Incorporated
FDA Decisions2 records
Approval1 INDICATIONS AND USAGE KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. ( 12.1 , 14 ) If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. ( 1 )(VRTX)FDA ↗
NDA207925Type 3 - New Dosage FormOrphan DrugPriorityOralGranule
Approval1 INDICATIONS AND USAGE KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. ( 12.1 , 14 ) If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. ( 1 )(VRTX)FDA ↗
NDA203188Type 1 - New Molecular EntityOrphan DrugPriorityOralTablet

Frequently asked questions

Which companies have sourced relationships with ivacaftor?

ivacaftor has dated SEC/FDA relationship records for VERTEX PHARMACEUTICALS INC / MA.

How many clinical trials involve ivacaftor?

BioSniper tracks 1 clinical trial involving ivacaftor.

What is ivacaftor's latest FDA decision?

The most recent tracked FDA decision for ivacaftor is Approval for 1 INDICATIONS AND USAGE KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies (14) ] . If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. ( 12.1 , 14 ) If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use. ( 1 ), dated March 17, 2015.