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Drug Name:


Generic Name and Formulations:
Ivacaftor 50mg, 75mg; per packet.

Vertex Pharmaceuticals

Therapeutic Use:


Treatment of cystic fibrosis (CF) in patients ≥2yrs who have one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.

Adults and Children:

<2yrs: not recommended. Oral granules should be mixed with 1 tsp (5mL) of soft-food or liquid (eg, yogurt, applesauce, milk, or juice) and completely consumed. Take with fat-containing food (eg, eggs, butter, peanut butter, cheese pizza, dairy products). 2–<6yrs: (<14kg): 50mg packet every 12 hours; (≥14kg): 75mg packet every 12 hours. ≥6yrs: 150mg tab every 12 hours. Concomitant strong CYP3A inhibitors: 150mg tab or 50mg packet (<14kg) or 75mg packet (≥14kg) twice weekly. Concomitant moderate CYP3A inhibitors: 150mg tab or 50mg packet (<14kg) or 75mg packet (≥14kg) once daily. Moderate hepatic impairment: 150mg tab or 50mg packet (<14kg) or 75mg packet (≥14kg) once daily. Severe hepatic impairment: 150mg tab or 1 packet once daily or less frequently.


If genotype is unknown, use an FDA-cleared CF mutation test to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions. Assess ALT/AST levels prior to initiating therapy, every 3 months during the first year of treatment, and annually thereafter. History of ALT/AST elevations: monitor LFTs more frequently. If increased ALT/AST levels develop, monitor closely until abnormalities resolved. Interrupt dosing if ALT/AST is >5xULN; after resolution, consider restarting. Perform baseline and follow-up eye exams. Hepatic impairment. Severe renal impairment or ESRD. Pregnancy. Nursing mothers.


Concomitant strong CYP3A inducers (eg, rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, St. John’s Wort): not recommended. Potentiated by strong CYP3A inhibitors (eg, ketoconazole, itraconazole, posaconazole, voriconazole, telithromycin, clarithromycin) and moderate CYP3A inhibitors (eg, fluconazole, erythromycin). May be potentiated by grapefruit and Seville oranges; avoid. Potentiates CYP3A and/or P-gp substrates (eg, digoxin, cyclosporine, tacrolimus).

See Also:


Pharmacological Class:

Cystic fibrosis transmembrane conductance regulator (CFTR) potentiator.

Adverse Reactions:

Headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, dizziness.

Generic Availability:


How Supplied:

Tabs—56, 60; Oral granules—56

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