Lumacaftor with ivacaftor

How it works

Lumacaftor improves the processing and trafficking of the defective CFTR protein to the cell surface, while ivacaftor potentiates the gating of the CFTR channel, together increasing chloride transport.

Prescribing in practice

• Lumacaftor is a strong inducer of CYP3A and significantly affects many co-administered drugs, including reducing the efficacy of hormonal contraceptives, so review all concomitant medication carefully.
• Respiratory adverse events such as chest tightness and dyspnoea can occur on initiation, particularly in those with lower lung function; monitor liver function.
• Ophthalmological assessment for cataracts is advised in paediatric patients.

Monitoring

Monitor liver transaminases before and during treatment, respiratory status on initiation, and perform eye examinations in children.

Counselling the patient

• Take with fat-containing food and avoid grapefruit products.
• Hormonal contraception may not be reliable with this medicine, so discuss alternative contraception.
• Report any chest tightness, breathlessness or signs of liver problems such as jaundice.

Evidence & guidelines

Lumacaftor with ivacaftor is supported by registration trials and NICE managed access showing modest lung-function improvement and reduced exacerbations in eligible patients.