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Orkambi(lumacaftor)
Orkambi (lumacaftor) is a small molecule pharmaceutical. Lumacaftor was first approved as Orkambi on 2015-07-02. It is used to treat cystic fibrosis in the USA. It has been approved in Europe to treat cystic fibrosis. It is known to target cystic fibrosis transmembrane conductance regulator.
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Commercial
Therapeutic Areas
Therapeutic Area
MeSH
digestive system diseasesD004066
respiratory tract diseasesD012140
hereditary congenital and neonatal diseases and abnormalitiesD009358
Trade Name
FDA
EMA
Combinations
Orkambi
Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
Ivacaftor
+
Lumacaftor
Tradename
Company
Number
Date
Products
ORKAMBIVertex PharmaceuticalsN-206038 RX2015-07-02
2 products, RLD, RS
ORKAMBIVertex PharmaceuticalsN-211358 RX2018-08-07
3 products, RLD, RS
Labels
FDA
EMA
Brand Name
Status
Last Update
orkambiNew Drug Application2020-05-11
Indications
FDA
EMA
Indication
Ontology
MeSH
ICD-10
cystic fibrosisEFO_0000390D003550E84
Agency Specific
FDA
EMA
Expiration
Code
IVACAFTOR / LUMACAFTOR, ORKAMBI, VERTEX PHARMS INC
2029-09-02ODE-408
2025-09-02NPP, NS
2025-08-07ODE-195
2023-09-28ODE-123
Patent Expiration
Patent
Expires
Flag
FDA Information
Ivacaftor / Lumacaftor, Orkambi, Vertex Pharms Inc
89936002030-12-11DP
85075342030-09-20DS, DP
87163382030-09-20DPU-1718, U-1910, U-2396, U-3426
91926062029-09-29DPU-1912, U-2397, U-3428
106464812029-08-13DP
115649162029-08-13U-3526, U-3529
88467182029-07-02U-1717, U-1908, U-2375, U-3427
86531032028-12-04DP
91505522028-12-04U-1908, U-2375, U-3427
100765132028-12-04DPU-2411
105973842028-12-04DS, DPU-2777, U-2778, U-3430
110520752028-12-04DPU-3181
83242422027-08-05U-1311, U-1911, U-2374, U-3424
74951032027-05-20DS, DP
84102742026-12-28DP
87542242026-12-28DS, DP
96701632026-12-28DPU-1911, U-2376, U-3429
99313342026-12-28DPU-2276, U-2376, U-3429
79730382026-11-08U-1973, U-2374, U-3424
87419332026-11-08U-1717, U-1909, U-2374, U-3424
92169692026-11-08DP
ATC Codes
R: Respiratory system drugs
— R07: Other respiratory system products in atc
— R07A: Other respiratory system products in atc
— R07AX: Other respiratory system products in atc
— R07AX30: Ivacaftor and lumacaftor
HCPCS
No data
Clinical
Clinical Trials
26 clinical trials
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Indications Phases 4
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Cystic fibrosisD003550EFO_0000390E845361924
Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Lung diseasesD008171EFO_0003818J98.4——1——1
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Long qt syndromeD008133HP_0001657I45.81—1———1
Indications Phases 1
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Hepatic insufficiencyD0485501————1
Healthy volunteers/patients—1————1
Indications Without Phase
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Glucose intoleranceD018149HP_0000833R73.03————11
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameLUMACAFTOR
INNlumacaftor
Description
Lumacaftor is an aromatic amide obtained by formal condensation of the carboxy group of 1-(2,2-difluoro-1,3-benzodioxol-5-yl)cyclopropane-1-carboxylic acid with the aromatic amino group of 3-(6-amino-3-methylpyridin-2-yl)benzoic acid. Used for the treatment of cystic fibrosis. It has a role as a CFTR potentiator and an orphan drug. It is a member of benzoic acids, a member of pyridines, an aromatic amide, a member of cyclopropanes, a member of benzodioxoles and an organofluorine compound.
Classification
Small molecule
Drug classcystic fibrosis transmembrane regulator (CFTR) protein modulators
Image (chem structure or protein)
Structure (InChI/SMILES or Protein Sequence)
Cc1ccc(NC(=O)C2(c3ccc4c(c3)OC(F)(F)O4)CC2)nc1-c1cccc(C(=O)O)c1
Identifiers
PDB—
CAS-ID936727-05-8
RxCUI1655922
ChEMBL IDCHEMBL2103870
ChEBI ID—
PubChem CID16678941
DrugBankDB09280
UNII IDEGP8L81APK (ChemIDplus, GSRS)
Target
Agency Approved
No data
Alternate
CFTR
CFTR
Organism
Homo sapiens
Gene name
CFTR
Gene synonyms
ABCC7
NCBI Gene ID
1080
Protein name
cystic fibrosis transmembrane conductance regulator
Protein synonyms
ATP-binding cassette sub-family C member 7, cAMP-dependent chloride channel, Channel conductance-controlling ATPase, cystic fibrosis transmembrane conductance regulating, cystic fibrosis transmembrane conductance regulator (ATP-binding cassette sub-family C, member 7)
Uniprot ID
Q2I102
Mouse ortholog
Cftr (12638)
cystic fibrosis transmembrane conductance regulator (Q9JKQ6)
Variants
Clinical Variant
Identifier
Target mutation
Effect
Evaluation
Status
VCV000007105CFTR, 1521_1523del, Phe508delPathogenic2004-03-031A
Financial
Orkambi - Vertex Pharmaceuticals
$
€
£
₣
Mock data
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Tabular view
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 1,758 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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2,767 adverse events reported
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