|
gptkbp:instance_of
|
gptkb:chemical_compound
|
|
gptkbp:bfsLayer
|
4
|
|
gptkbp:bfsParent
|
gptkb:Orkambi
|
|
gptkbp:activities
|
corrects CFTR protein folding
|
|
gptkbp:approves
|
gptkb:FDA
|
|
gptkbp:can_be_used_with
|
gptkb:ivacaftor
|
|
gptkbp:clinical_trial
|
Phase 3
long-term treatment
TRAFFIC
TRANSPORT
|
|
gptkbp:contraindication
|
severe liver impairment
|
|
gptkbp:developed_by
|
gptkb:Vertex_Pharmaceuticals
|
|
gptkbp:education
|
necessary for understanding treatment
|
|
gptkbp:effective_date
|
gptkb:2015
|
|
gptkbp:excretion
|
urine
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
lumacaftor
|
|
gptkbp:indication
|
homozygous F508del mutation in CFTR gene
|
|
gptkbp:ingredients
|
C24 H26 Cl F2 N3 O2 S
|
|
gptkbp:interacts_with
|
with strong CY P3 A inducers
with strong CY P3 A inhibitors
|
|
gptkbp:is_atype_of
|
R07 A X03
|
|
gptkbp:is_used_for
|
gptkb:cystic_fibrosis
|
|
gptkbp:lifespan
|
about 24 hours
|
|
gptkbp:manager
|
oral
|
|
gptkbp:marketed_as
|
gptkb:legislation
gptkb:Australia
gptkb:United_States
gptkb:Orkambi
gptkb:Native_American_tribe
|
|
gptkbp:pharmacokinetics
|
metabolized by CY P3 A
|
|
gptkbp:population
|
adults and children over 2 years
|
|
gptkbp:regulatory_compliance
|
gptkb:Health_Canada
gptkb:European_Medicines_Agency
Australian Therapeutic Goods Administration
|
|
gptkbp:research_areas
|
personalized medicine
drug delivery systems
genetic therapies
clinical outcomes in CF
|
|
gptkbp:research_focus
|
gptkb:CFTR_modulators
|
|
gptkbp:safety_features
|
generally well tolerated
|
|
gptkbp:side_effect
|
fatigue
headache
nausea
diarrhea
rash
liver enzyme elevation
respiratory symptoms
gastrointestinal symptoms
|
|
gptkbp:targets
|
gptkb:cystic_fibrosis
|
|
gptkbp:treatment
|
improve quality of life
improve lung function
reduce pulmonary exacerbations
with ivacaftor
|
|
gptkbp:type_of
|
1005290-37-4
|
|
gptkbp:type_of_care
|
important for treatment efficacy
|
|
gptkbp:weight
|
471.00 g/mol
|