What Medications Are Used to Treat Cystic Fibrosis?

Cystic fibrosis (CF) affects the lungs the most, but also impacts the digestive system, including the pancreas, liver, intestines, and kidneys. CF may also impact the ears, nose and sinuses, sex organs, and the bones and joints. Because CF attacks so many organs in the body and causes persistent lung infections, many medications are usually needed to manage the disease.

Medications used to treat cystic fibrosis

The CF Foundation recommends the following order of inhaled medications:

• Bronchodilator
• Mucus thinner (mucolytic)
• Aerosolized antibiotic

These drugs are used in combination with airway clearance techniques in a specific order designed to move mucus out of the lungs. Other medications that people with CF often take include oral and IV antibiotics, CFTR modulators, and ibuprofen, in addition to nutritional supplements such as pancreatic enzymes and vitamins.

Bronchodilators

Bronchodilators make breathing easier by opening the airways so that mucus can be cleared out. The person with CF breathes through a device that delivers air-borne medicine directly into their airways. Albuterol and levabuterol are common drugs delivered through a nebulizer or inhaler.

Mucus thinners/mucolytics

Mucus thinners or mucolytics are drugs that make the thick, sticky mucus easier to move out of the lungs and sinuses. Most people with CF inhale their mucus thinners, but the drugs also come in tablet form for people without CF. Commonly prescribed mucus thinners for CF include hypertonic saline, dornase alfa (Pulmozyme), and mannitol (Bronchitol).^1,2

Antibiotics

People with CF are especially prone to respiratory infections, so many take antibiotics daily to help control those bacteria. These drugs are delivered in pill form or are inhaled. Intravenous (IV) antibiotics are used during exacerbations. Controlling the organisms that cause lung infections helps improve lung function, making it easier to breathe, and helps reduce the chances of an exacerbation.

Commonly used antibiotics for people with CF include azithromycin, tobramycin (Tobi, Bethkis, TobiPodhaler), and aztreonam (Cayston).^1,2,4 Because such large, continual doses of antibiotics must be used, many people with CF develop antibiotic resistance. This means the drugs stop killing or weakening the bacteria. Antibiotic resistance makes it harder to control lung infections over the long term. It is important to work with a CF healthcare team to find the best way to treat infections and reduce antibiotic resistance.

CFTR modulators

A class of drugs for CF called CFTR modulators target specific defects in the CFTR protein so that the protein can work properly. CFTR modulators do not fully restore chloride flow, but they do improve the flow enough to relieve CF symptoms. There are currently three different types of CFTR modulators and each CFTR modulator is designed to correct specific problems caused by the mutated CFTR protein.

Not all CFTR modulators work for all CF patients because different genetic mutations cause different types of CFTR protein defects.These drugs are named elexacaftor/tezacaftor/ivacaftor (Trikafta), ivacaftor (Kalydeco), lumacaftor/ivacaftor (Orkambi), and tezacaftor/ivacaftor (Symdeko).³

Pancreatic enzymes

Even though pancreatic enzymes are supplements, they are integral to CF and are usually grouped in as medications. Pancreatic insufficiency (PI) is a significant issue for the majority of people living with CF. Left untreated, it can lead to growth failure, weight loss, abdominal bloating, foul-smelling stools, or diarrhea. Using pancreatic enzyme replacement therapy (PERT) helps to alleviate these symptoms.⁵

Ibuprofen

The anti-inflammatory drug Ibuprofen (Motrin, Advil) is recommended for certain CF patients between 6 and 17 years old. Studies have shown that daily doses of ibuprofen slow the loss of lung function in children and teens with CF. The amount taken must be calculated by a doctor to get the right amount. Too low a dose and the child won’t get the benefit, and too high a dose is dangerous.^1,2

Ibuprofen is not recommended for those 18 and older since research does not prove that it reduces exacerbations or slows the loss of lung function.²