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: Tezacaftor (TEZ) is a CFTR modulator that facilitates the folding and stability of CFTR and increases its processing and intracellular transport to the cell membrane. Ivacaftor (IVA) is a CFTR potentiator that increases the probability that these chlorine channels remain open at the cell surface. Symdeko is copackaged as TEZ 100 milligram (mg)/IVA 150 mg and a separate tablet of IVA 150 mg.

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 TEZ-IVA in addition to standard care effective in treating patients with CF who are homozygous for the F508del mutation or patients who are compound heterozygous for the F508del mutation and another CF mutation?

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

  • Is TEZ-IVA safe?

  • Have definitive patient selection criteria been identified for the use of TEZ-IVA?

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