Kaftrio

Product Information:

Kaftrio is a prescription medicine used to treat cystic fibrosis (CF) — a genetic disorder that causes thick, sticky mucus in the lungs and digestive system. It contains three active drugs:

• Ivacaftor

• Tezacaftor

• Elexacaftor
  These work together to improve the function of the defective CFTR protein that causes CF.European Medicines Agency (EMA)+1

Who it’s for:

• Patients aged 2 years and older with CF who have at least one responsive CFTR gene mutation (often the F508del mutation).European Medicines Agency (EMA)

How it works:

• Tezacaftor and elexacaftor help more CFTR protein reach the cell surface.

• Ivacaftor enhances the activity of the CFTR protein once it’s there.
  This combination improves mucus clearance, lung function, and other CF symptoms.European Medicines Agency (EMA)

Uses:

Kaftrio is not a cure, but it targets the underlying cause of CF in people with specific gene mutations. Main benefits include:

• Improved lung function

• Fewer pulmonary exacerbations

• Thinner mucus and easier breathing
  It is intended as a long‑term treatment.

Dosage:

Important: Dosage must be tailored by a healthcare professional experienced in CF care

Typical dosing patterns:

Ages 6 years and older

• Morning: Kaftrio tablets

• Evening: (≈12 h later): Ivacaftor alone
  Depending on weight and age, your doctor will prescribe the correct strength and quantity.

• Younger children (2–<6 years):

• Kaftrio granules are available and mixed with soft food or milk; taken with a fat‑containing meal.

Key points about dosing:

• Take with fat‑containing food to aid absorption.

• Avoid grapefruit products during treatment (can increase side effects).

Dose adjustments:

• May be needed if you’re on other medications that affect liver metabolism (CYP3A inhibitors).

• In liver impairment, your doctor may change the dose.European Medicines Agency (EMA)

Side Effects:

• Headache

• Diarrhoea

• Upper respiratory infections (nose/throat)

• Rash
  These are generally mild to moderate but tell your doctor if bothersome

• Liver enzyme increases (should be monitored with periodic blood tests)

• Elevated blood pressure

• Cataracts (especially in children — eye checks may be recommended)

• Psychological symptoms: anxiety, low mood, sleep disturbance, or changes in behaviour have been reported, though uncommon