drug · small molecule

KALYDECO

updated 3mo ago

/ EYE-və-KAF-tər /

by $VERX Vertex, Inc.🔗 ChEMBL

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Small molecule drug with a maximum clinical stage of Approval (across all indications), with 3 approved and 8 investigational indications.

targets

Cystic fibrosis transmembrane conductance regulator

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Trials studying KALYDECO

phase1Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosisrecruitingn=255
phase2A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CFrecruitingn=192
phase2Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansioncompletedn=40
phase2The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftorcompletedn=40
phase1Bioequivalence and Food Effect Bioavailability Study of Lumacaftor Film-Coated Tabletscompletedn=39
phase2A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allelecompletedn=17
phase2Gut Imaging for Function & Transit in Cystic Fibrosis Study 2completedn=12

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brand

KALYDECO

trade names

Ivacaftor component of orkambi, Ivacaftor component of symkevi, Ivacaftor component of trikafta, Kalydeco

generic

IVACAFTOR

MOA

12.1 Mechanism of Action Ivacaftor is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. Ivacaftor facilitates increased chloride transport by potentiating the channel open probability (or gating) of CFTR protein located at the cell surface. The overall level of ivacaftor-mediated CFTR chloride transport is dependent on the amount of CFTR protein at the cell surface and how responsive a particular mutant CFTR protein is to ivacaftor potentiation. CFTR Chloride Transport Assay in Fischer Rat Thyroid (FRT) cells expressing mutant CFTR The chloride transport response of mutant CFTR protein to ivacaftor was determined in Ussing chamber electrophysiology studies using a panel of FRT cell lines transfected with individual CFTR mutations. Ivacaftor increased chloride transport in FRT cells expressing CFTR mutations that result in CFTR protein being delivered to the cell surface. The in vitro CFTR chloride transport response threshold was designated as a net increase of at least 10% of normal over baseline because it is predictive or reasonably expected to predict clinical benefit. For individual mutations, the magnitude of the net change over baseline in CFTR-mediated chloride transport in vitro is not correlated with the magnitude of clinical response. A patient must have at least one CFTR mutation responsive to ivacaftor to be indicated. Note that splice site mutations cannot be studied in the FRT assay. Evidence of clinical efficacy exists for non-canonical splice mutations 2789+5G→A , 3272-26A→G , 3849+10kbC→T , 711+3A→G and E831X and these are listed in Table 3 below [see also Clinical Studies (14.4) ] . The G970R mutation causes a splicing defect resulting in little-to-no CFTR protein at the cell surface that can be potentiated by ivacaftor [see Clinical Studies (14.2) ] . Ivacaftor also increased chloride transport in cultured human bronchial epithelial (HBE) cells derived from CF

indication

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 )

Data sources

ClinicalTrials.gov · OpenTargets

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KALYDECO

updated 3mo ago

/ EYE-və-KAF-tər /

by $VERX Vertex, Inc.🔗 ChEMBL

community read0 votes

🐶 often sniffed alongside

top 8

Small molecule drug with a maximum clinical stage of Approval (across all indications), with 3 approved and 8 investigational indications.

targets

Cystic fibrosis transmembrane conductance regulator

all catalysts(0)

nothing on the calendar yet

Trials studying KALYDECO

phase1Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosisrecruitingn=255
phase2A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CFrecruitingn=192
phase2Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansioncompletedn=40
phase2The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftorcompletedn=40
phase1Bioequivalence and Food Effect Bioavailability Study of Lumacaftor Film-Coated Tabletscompletedn=39
phase2A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allelecompletedn=17
phase2Gut Imaging for Function & Transit in Cystic Fibrosis Study 2completedn=12

News(0)

no news has mentioned this drug yet

brand

KALYDECO

trade names

Ivacaftor component of orkambi, Ivacaftor component of symkevi, Ivacaftor component of trikafta, Kalydeco

generic

IVACAFTOR

MOA

indication

Data sources

ClinicalTrials.gov · OpenTargets

last refreshed 55m ago

Found something off?

comments(0)

5-min edit window · permanent after that

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KALYDECO

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Trials studying KALYDECO

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KALYDECO

🐶 often sniffed alongside

targets

all catalysts(0)

Trials studying KALYDECO

News(0)

Report a data issue

comments(0)