Medication	Dosage	Effects	Adverse effects and contraindications	Monitoring and end points
Cystic fibrosis transmembrane conductance regulator protein modulators
Ivacaftor (Kalydeco; for patients 1 month or older) Lumacaftor/ivacaftor (Orkambi; for patients 1 year or older) Tezacaftor/ivacaftor (Symdeko; for patients 6 years or older) Elexacaftor/tezacaftor/ivacaftor (Trikafta; for patients 2 years or older)	Varies based on drug used and patient weight Eligibility based on specific mutations	Improved lung function and QOL Decreased pulmonary exacerbations Reduced sweat chloride levels Improved body mass index	Gastrointestinal issues Elevated findings on LFTs (liver enzymes/bilirubin) Body aches Headaches and dizziness Rash	Monitor liver function regularly Monitor for cataracts Consider drug-drug interactions; consult https://www.vertextreatmentshcp.com Advise caution when used with substances that affect LFT results (e.g., alcohol, other medications)
Inhaled mucolytics
Dornase alfa (Pulmozyme)	2.5 mg, once or twice per day	Improved lung function and QOL Decreased use of antibiotics, pulmonary exacerbations and hospitalizations	Voice alteration and throat problems Hemoptysis	—
Hypertonic saline	4 mL of 7% saline	Decreased pulmonary exacerbations	Cough and bronchospasm	—
Inhaled antibiotics
Tobramycin (inhalation solution or TOBI Podhaler)	1 vial of tobramycin (300 mg) inhalation solution, twice per day or 4 TOBI Podhaler capsules, twice per day	Improved lung function and QOL Decreased pulmonary exacerbations, use of intravenous antibiotics, and hospitalization	Tinnitus Voice alteration and throat problems Significant increase in cough that lasts for treatment duration with TOBI Podhaler	Monitor blood urea nitrogen/creatinine ratio at random intervals if kidney insufficiency is present Refer for audiology evaluation in young children or if there is a concern for hearing loss
Aztreonam (nebulizer solution)*	75 mg, three times per day (3-minute duration) Separate doses by at least 4 hours	Improved lung function and QOL Prolonged time to exacerbation	Allergy Bronchospasm and wheezing Cough	—
Gastrointestinal medications
Pancreatic enzymes: pancrelipase (ZenPep, Creon, Pancreaze, Pertzye, Ultresa, Viokase)	Varies based on drug and patient weight Requires brands specific to cystic fibrosis	Improved absorption of nutrients, including vitamins and minerals from gastrointestinal tract Maintenance of healthy body mass index (≥ 50th percentile) Decreased abdominal pain Improved texture and frequency of stool	Abdominal pain Flatulence Fibrosing colonopathy (rare)	Monitor for signs of malabsorption, such as stools that are more frequent, foul smelling, or have a change in consistency
Ursodiol	10 to 15 mg per kg, divided, twice per day; typical maximum dosage: 300 mg twice per day	Improved liver function laboratory values	Primary sclerosing cholangitis Contraindicated with complete biliary obstruction	Manage in conjunction with hepatologist Monitor liver function; perform annual liver Doppler ultrasonography
Anti-inflammatories
Ibuprofen (for patients older than 6 years)	Based on individual pharmacokinetics, maximum dosage: 1,600 mg twice per day	Preservation of lung function 33% less decline FEV1	Long-term kidney function safety concerns Risk of ulcers and gastrointestinal bleeding with high doses	Consider monitoring kidney function
Azithromycin (for patients older than 6 years)	25 to 40 kg: 250 mg MWF > 40 kg: 500 mg MWF	Improved lung function Decreased pulmonary exacerbations	Elevated LFTs (more than three times the upper limit of normal), contraindicated to therapy QTc prolongation/torsades de pointes Nausea and diarrhea	Electrocardiography before initiation, then 2 to 3 months after starting and 2 to 3 months after any dosage increase Sputum acid-fast bacillus test annually