|
gptkbp:instance_of
|
gptkb:pharmaceuticals
|
|
gptkbp:approves
|
gptkb:2015
gptkb:FDA
|
|
gptkbp:availability
|
limited in some countries
|
|
gptkbp:can_be_combined_with
|
gptkb:ivacaftor
|
|
gptkbp:class
|
gptkb:CFTR_modulators
|
|
gptkbp:clinical_trial
|
Phase 3
NCT02122715
NCT02139524
NCT02139537
NCT02140024
NCT02140037
|
|
gptkbp:clinical_use
|
long-term treatment
|
|
gptkbp:contraindication
|
liver disease
hypersensitivity to components
|
|
gptkbp:dosage_form
|
gptkb:tablet
|
|
gptkbp:effective_date
|
gptkb:2015
|
|
gptkbp:financial_support
|
available
|
|
gptkbp:formulation
|
combination therapy
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Orkambi
|
|
gptkbp:indication
|
homozygous F508del mutation
|
|
gptkbp:ingredients
|
gptkb:ivacaftor
gptkb:lumacaftor
|
|
gptkbp:invention
|
gptkb:Vertex_Pharmaceuticals
|
|
gptkbp:manufacturer
|
gptkb:Vertex_Pharmaceuticals
|
|
gptkbp:marketed_as
|
gptkb:Canada
gptkb:European_Union
gptkb:United_States
|
|
gptkbp:mechanism_of_action
|
corrects CFTR protein folding
|
|
gptkbp:patient_population
|
homozygous F508del mutation carriers
|
|
gptkbp:pharmacokinetics
|
excreted in urine
improves quality of life
metabolized by liver
improves lung function
reduces pulmonary exacerbations
|
|
gptkbp:price
|
high
|
|
gptkbp:provides_guidance_on
|
recommended for specific patients
|
|
gptkbp:regulatory_compliance
|
prescription only
|
|
gptkbp:research_focus
|
cystic fibrosis treatment
|
|
gptkbp:research_output
|
reduced hospitalizations
improved lung function
enhanced overall health status
improved weight gain
reduced CF-related complications
|
|
gptkbp:route_of_administration
|
oral
|
|
gptkbp:side_effect
|
fatigue
headache
nausea
diarrhea
rash
insomnia
shortness of breath
liver problems
elevated liver enzymes
respiratory symptoms
|
|
gptkbp:target_audience
|
adults
children over 2 years
|
|
gptkbp:used_for
|
gptkb:cystic_fibrosis
|
|
gptkbp:bfsParent
|
gptkb:Vertex_Pharmaceuticals
|
|
gptkbp:bfsLayer
|
4
|