Europe — EMA
Orkambi (Lumacaftor, Ivacaftor)
GNH USA supplies Orkambi (Lumacaftor/Ivacaftor), a CFTR modulator for cystic fibrosis patients with the F508del mutation. Request a quote for global supply.
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What is Lumacaftor, Ivacaftor?
Lumacaftor/Ivacaftor, the active ingredient in Orkambi, is a CFTR modulator presented as a 400 mg/250 mg oral tablet for oral use. It is indicated for cystic fibrosis patients who have two copies of the F508del mutation. GNH USA supplies Orkambi as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, pharmacies and clinics worldwide.
What is Lumacaftor, Ivacaftor used for?
Orkambi is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator used in patients with the F508del mutation. - Cystic fibrosis (F508del homozygous, ≥12 years): improves lung function and reduces pulmonary exacerbations. - Cystic fibrosis (F508del homozygous, ≥12 years): lowers sweat chloride concentrations, reflecting enhanced CFTR activity. - Cystic fibrosis (F508del homozygous, ≥12 years): contributes to better nutritional status and weight gain.
What are the side effects of Lumacaftor, Ivacaftor?
Orkambi may cause a range of adverse reactions, most of which are mild to moderate.
Common Side Effects: - Headache: mild to moderate intensity. - Upper respiratory tract infection: symptoms of congestion or sore throat. - Nausea: occasional stomach upset. - Diarrhea: loose stools, generally self‑limiting. - Elevated liver enzymes: transient increase in ALT/AST. - Rash: mild skin irritation or erythema.
Serious or Rare Side Effects: - Hepatotoxicity: significant elevation of liver enzymes requiring monitoring. - Severe respiratory events: bronchospasm or worsening of pulmonary status. - Allergic reactions: anaphylaxis or severe skin reactions such as Stevens‑Johnson syndrome. - Vision changes: rare reports of cataract formation. - Cardiac arrhythmias: QT prolongation in susceptible individuals.
What precautions apply to Lumacaftor, Ivacaftor?
Before initiating Orkambi, consider the following precautions. - Liver function monitoring: assess baseline ALT/AST and repeat periodically. - Drug–enzyme interactions: review concomitant CYP3A4 substrates or inhibitors. - Pregnancy and lactation: weigh potential benefits against unknown fetal risks; consult specialist. - Respiratory status: monitor for worsening symptoms, especially in severe disease. - Age restriction: approved for patients aged 12 years and older with the specified genotype. Store at ambient temperature below 25°C: keep in the original container, protect from moisture and direct sunlight.
What does Lumacaftor, Ivacaftor interact with?
Orkambi can interact with several medicines, requiring careful management.
Major Interactions (Avoid): - Strong CYP3A4 inducers (e.g., rifampin, carbamazepine): may markedly reduce exposure. - Strong CYP3A4 inhibitors (e.g., ketoconazole, clarithromycin): can increase systemic levels and toxicity.
Moderate Interactions (Monitor Closely): - Moderate CYP3A4 inhibitors (e.g., fluconazole): may modestly raise concentrations. - Medications affecting gastric pH (e.g., proton pump inhibitors): could alter absorption. - Anticoagulants (e.g., warfarin): monitor INR as changes in liver metabolism may occur.
