drug · small molecule

ORKAMBI

updated 3mo ago

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

all catalysts(0)

nothing on the calendar yet

Trials studying ORKAMBI

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

ORKAMBI

trade names

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

generic

IVACAFTOR

MOA

12.1 Mechanism of Action The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. The F508del mutation results in protein misfolding, causing a defect in cellular processing and trafficking that targets the protein for degradation and therefore reduces the quantity of CFTR at the cell surface. The small amount of F508del-CFTR that reaches the cell surface is less stable and has low channel-open probability (defective gating activity) compared to wild-type CFTR protein. Lumacaftor improves the conformational stability of F508del-CFTR, resulting in increased processing and trafficking of mature protein to the cell surface. Ivacaftor is a CFTR potentiator that facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein at the cell surface. In vitro studies have demonstrated that both lumacaftor and ivacaftor act directly on the CFTR protein in primary human bronchial epithelial cultures and other cell lines harboring the F508del-CFTR mutation to increase the quantity, stability, and function of F508del-CFTR at the cell surface, resulting in increased chloride ion transport. In vitro responses do not necessarily correspond to in vivo pharmacodynamic response or clinical benefit.

indication

1 INDICATIONS AND USAGE ORKAMBI is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene. ORKAMBI is a combination of ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, and lumacaftor, indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene. ( 1 ) Limitations of Use: The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation. ( 1 ) Limitations of Use The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation .

Data sources

ClinicalTrials.gov · OpenTargets

last refreshed 43m ago

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drug · small molecule

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ORKAMBI

updated 3mo ago

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 ORKAMBI

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

ORKAMBI

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 43m ago

Found something off?

comments(0)

5-min edit window · permanent after that

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ORKAMBI

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targets

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Trials studying ORKAMBI

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ORKAMBI

🐶 often sniffed alongside

targets

all catalysts(0)

Trials studying ORKAMBI

News(0)

Report a data issue

comments(0)