Lumacaftor

What it is used for

ORKAMBI is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene.

Side effects, and when to stop

The following adverse reactions are discussed in greater detail in other sections of the label:

• Use in Patients with Advanced Liver Disease
• Liver-related Events
• Hypersensitivity Reactions, Including Anaphylaxis
• Intracranial Hypertension
• Neuropsychiatric Events, Including Suicidal Thoughts and Behaviors
• Respiratory Events
• Effect on Blood Pressure
• Cataracts

Who should not take it

None.

Storing it

Store at 20°C - 25°C (68°F - 77°F); excursions permitted to 15°C - 30°C (59°F - 86°F).