What medications are used to treat cystic fibrosis?

From the Guidelines

Medications used to treat cystic fibrosis include CFTR modulators, mucolytics, bronchodilators, antibiotics, anti-inflammatory medications, pancreatic enzyme replacement therapy, and multivitamins. The treatment of cystic fibrosis is complex and multifaceted, requiring a comprehensive approach to manage the disease's various manifestations. For patients with cystic fibrosis, the use of inhaled tobramycin is strongly recommended for those 6 years of age and older with moderate to severe lung disease and persistent presence of P. aeruginosa in airway cultures, as it improves lung function and reduces exacerbations 1. Additionally, azithromycin is recommended for patients with CF and Pseudomonas aeruginosa persistently present in airway cultures to improve lung function and reduce exacerbations 1. Mucolytics like dornase alfa are also strongly recommended for patients with moderate to severe lung disease to improve lung function 1. Other key medications include:

• CFTR modulators such as Trikafta (elexacaftor/tezacaftor/ivacaftor), Symdeko (tezacaftor/ivacaftor), Orkambi (lumacaftor/ivacaftor), and Kalydeco (ivacaftor)
• Mucolytics like Pulmozyme (dornase alfa) and hypertonic saline (3-7%)
• Bronchodilators such as albuterol
• Antibiotics like tobramycin, aztreonam, and azithromycin
• Anti-inflammatory medications like ibuprofen
• Pancreatic enzyme replacement therapy (PERT) such as Creon or Zenpep
• Multivitamins, especially fat-soluble vitamins (A, D, E, K) Treatment is highly individualized based on specific mutations, symptoms, and complications, requiring regular monitoring and adjustment by a specialized CF care team.

From the FDA Drug Label

TOBI Podhaler is a prescription medicine used to treat people with cystic fibrosis who have a bacterial infection called Pseudomonas aeruginosa. The most frequently used other antibacterial drugs (any route of administration) were azithromycin, ciprofloxacin, and ceftazidime Consistent with the population of cystic fibrosis patients, the most frequently used concomitant medications included oral pancreatic enzyme preparations, mucolytics (especially dornase alfa), and selective β2-adrenoreceptor agonists.

The medications used to treat cystic fibrosis include:

• Tobramycin (INH): an antibacterial medicine used to treat Pseudomonas aeruginosa infection
• Azithromycin: an antibacterial drug
• Ciprofloxacin: an antibacterial drug
• Ceftazidime: an antibacterial drug
• Oral pancreatic enzyme preparations: to help with digestion
• Mucolytics (especially dornase alfa): to help thin mucus
• Selective β2-adrenoreceptor agonists: to help with breathing 2, 2, 2

From the Research

Cystic Fibrosis Treatment Drugs

• Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are a class of medications used to treat cystic fibrosis, including tezacaftor and ivacaftor 3.
• Ivacaftor is a CFTR potentiator approved for the treatment of CF patients with at least one copy of the G551D-CFTR mutation 4.
• Lumacaftor in combination with ivacaftor is used to treat patients with two copies of the F508del mutation 5.
• Other medications, such as correctors, are being developed to address the underlying cause of cystic fibrosis, including the p.Phe508del mutation 6.

Types of Cystic Fibrosis Medications

• CFTR potentiators, such as ivacaftor, which improve the function of defective CFTR proteins 4.
• CFTR correctors, such as VX-809, which improve the trafficking of defective CFTR proteins to the cell surface 6.
• Ion channel modifiers, which target alternative chloride channels or inhibit sodium absorption 7.
• Osmotically active agents, which help to thin mucus and improve mucociliary clearance 7.

Treatment Recommendations

• The Cystic Fibrosis Foundation recommends the use of CFTR modulators, such as ivacaftor and lumacaftor, for patients with specific mutations 5.
• Treatment decisions should be based on individual patient needs and mutation status 5.
• Clinical trials have shown that CFTR modulators can improve lung function and reduce pulmonary exacerbations in patients with cystic fibrosis 3, 4.