NAPLEX (North American Pharmacist Licensure Examination) Practice Exam

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Which CFTR modulator is NOT approved for homozygous F508del mutation?

  1. Ivacaftor (Kalydeco)

  2. Lumacaftor/ivacaftor (Orkambi)

  3. Tezacaftor/ivacaftor (Symdeko)

  4. Elexacaftor/tezacaftor/ivacaftor (Trikafta)

The correct answer is: Ivacaftor (Kalydeco)

All of the other options, B, C, and D, are approved for homozygous F508del mutation. Option A, Ivacaftor, is actually approved for the G551D mutation and not for homozygous F508del mutation. This is an important distinction as each CFTR modulator is designed to treat specific genetic mutations in cystic fibrosis patients. Therefore, a patient with homozygous F508del mutation would not benefit from taking Ivacaftor (Kalydeco) and may require a different CFTR modulator treatment.

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