With Kaftrio being used for treatment of patients with Cystic Fibrosis, comes serious negative impact on patient’s psychological state. As of 25 February 2025, the MHRA received 300 reports of suspected psychological side effects linked to Kaftrio, out of over 9,000 UK users. Some cases involved patients with no previous mental health history. The frequency of these effects remains unclear, and monitoring is ongoing.
Some of these side effects which usually appear within the first 3 month included anxiety, low mood, sleep disturbance, poor concentration, and forgetfulness, Although the risk is low, patients should stay aware of any changes in mood or behavior and seek medical advice promptly if they notice anything unusual.
Advice for Healthcare Professionals
- Mental health changes may also stem from adjusting to improved physical health and quality of life.
- People with cystic fibrosis already have a higher risk of poor mental health.
- In children, psychological side effects may appear as persistent changes, look for behavior changes like increased disruptiveness.
- These symptoms can appear even in those with no mental health history.
- Discuss benefits vs. risks with the patient or caregiver.
- Consider stopping Kaftrio if psychological side effects occur.
The UK Summary of Product Characteristics (SmPC) and Patient Information Leaflet (PIL) for Kaftrio will be updated to include a warning about potential side effects affecting mood, sleep, concentration, and behavior.
Patients and caregivers will be advised to monitor for these symptoms and to contact a healthcare professional promptly if they occur. Or directly report to the yellow scheme.
Learn more: Kaftrio▼ (Ivacaftor, tezacaftor, elexacaftor): risk of psychological side effects - GOV.UK
