Lumacaftor/Ivacaftor (Orkambi)

For treating cystic fibrosis in patients 12 years or older and who have 2 copies of F5o8del mutations in the CFTR gene.

Approximately 50% of the 30,000 patients with cystic fibrosis in the US have 2 copies of the F5o8del mutation in the CFTR G and would be eligible to take this drug combination.

Phase 3 studies show a 3% improvement in lung function over placebo.

Dosage is 2 pills every 12 hours.

Side effects include: Shortness of breath, upper respiratory tract infection, nausea, diarrhea, rash, and increased menstrual abnormalities in women.

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