Lumacaftor
- Generic Name
- Lumacaftor
- Brand Names
- Orkambi
- Drug Type
- Small Molecule
- Chemical Formula
- C24H18F2N2O5
- CAS Number
- 936727-05-8
- Unique Ingredient Identifier
- EGP8L81APK
- Background
Lumacaftor is a drug used in combination with Ivacaftor as the fixed dose combination product Orkambi for the management of Cystic Fibrosis (CF) in patients aged 6 years and older. Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) prot...
- Indication
When used in combination with the drug lumacaftor as the product Orkambi, ivacaftor is indicated for the management of CF in patients aged one 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 ...
- Associated Conditions
- Cystic Fibrosis (CF)
- Associated Therapies
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The impact of the COVID-19 pandemic and the changing landscape of CF on the cASPerCF trial
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