Health Problem: Cystic fibrosis (CF) is an autosomal recessive genetic disease due to mutations in the cystic fibrosis transmembrane receptor (CFTR) gene. The basic defect results in poor chloride and bicarbonate transport resulting in dehydration of secretions in the lungs, pancreas, and other organs. The bronchi and lungs are affected by recurrent bronchitis and bronchopneumonia, with progressive deterioration in pulmonary function, which remains the major cause of morbidity and mortality and reduced life expectancy.

Technology Description: The combined lumacaftor (LUM)-ivacaftor (IVA) oral tablet contains 200 milligrams (mg) of LUM and 125 mg of IVA. LUM is a CFTR corrector that facilitates the folding and stability of CFTR and increases its processing and intracellular transport to the cell membrane. IVA is a CFTR potentiator that increases the probability that these chlorine channels remain open at the cell surface.

Controversy: Patients with CF are cared for with multimodality approaches that include pancreatic enzyme replacement therapy, antipseudomonal antibiotics, enhancement of mucociliary clearance with medication and chest physiotherapy, infection control, nutrition, and diagnosis and management of CF-related diabetes. All of these therapies address the symptoms of CF rather than the underlying CFTR malfunction.

Key Questions:

  • Is LUM-IVA in addition to standard care effective in treating CF in patients ≥ 6 years of age who are homozygous for the F508del mutation in the CFTR gene?

  • How does LUM-IVA compare with standard care or clinical alternatives?

  • Have definitive patient selection criteria been identified for LUM-IVA?

  • Is LUM-IVA safe?

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