Tezacaftor/ivacaftor

(Symdeko®)

Symdeko®

Drug updated on 10/29/2024

Dosage FormTablet (oral; tezacaftor/ivacaftor and ivacaftor: 50 mg/75 mg and 75 mg); Tablet (oral; tezacaftor/ivacaftor and ivacaftor: 100 mg/150 mg and 150 mg)
Drug ClassCFTR potentiators and correctors
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.

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Summary
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  • This summary is based on the review of five systematic review(s)/meta-analysis(es). [1-5]
  • Elexacaftor-Tezacaftor-Ivacaftor (Trikafta) Triple Therapy significantly improved pulmonary function (FEV1) and quality of life (QoL) in cystic fibrosis (CF) patients aged 12 years and older with F508del variants, leading to reduced exacerbation rates.
  • Ivacaftor (IVA) Monotherapy resulted in notable weight gain in children and adults with at least one G551D mutation, while no significant body mass index (BMI) changes were observed in adults with the R117H mutation or children homozygous for F508del receiving IVA with Tezacaftor.
  • Combination Therapies demonstrated varying effectiveness, with Elexacaftor-Tezacaftor-Ivacaftor showing superior improvements in QoL and lung function compared to both Lumacaftor-Ivacaftor and Tezacaftor-Ivacaftor, which had moderate improvements.
  • Common Adverse Effects of Elexacaftor-Tezacaftor-Ivacaftor include headache and rash, while Lumacaftor-Ivacaftor is associated with early transient breathlessness and increased blood pressure.
  • Elexacaftor-Tezacaftor-Ivacaftor demonstrated comparable adverse events to placebo or control, whereas Tezacaftor-Ivacaftor reported fewer adverse events compared to Lumacaftor-Ivacaftor.
  • Studies included individuals aged 12 years and older, with significant benefits observed in the F508del mutation cohort from Elexacaftor-Tezacaftor-Ivacaftor, and notable weight gain in individuals with at least one G551D mutation from Ivacaftor; effectiveness and safety for younger populations (<12 years) were not well established.