Summary

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• Lumacaftor/ivacaftor (Orkambi) is indicated for the treatment of cystic fibrosis in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. It has shown improvements in quality of life scores, particularly in the respiratory domain, and relative changes in forced expiratory volume when compared to placebo.
• Six studies provided comparative information on Orkambi's safety and effectiveness against other drugs used to treat cystic fibrosis.
• Compared to dual therapies like lumacaftor/ivacaftor, triple therapies such as elexacaftor-tezacaftor-ivacaftor have shown significant impact, especially among those with specific CF mutations. They also resulted in fewer pulmonary exacerbations, which is a critical outcome for patients with this condition.
• Despite being generally well tolerated, lumacaftor/ivacaftor was associated with a higher frequency of adverse events, including respiratory-related issues leading to discontinuation. This raises concerns regarding its long-term use due to potential side effects like early transient breathlessness and increases in blood pressure not reported with another regimen.
• Significant weight gain and improvements were noted, specifically among children using ivacaftor or adults over 12 years old using a different combination, indicating positive effects on nutritional status, which is crucial for managing cystic fibrosis.
• The efficacy varies based on specific CFTR mutation type, highlighting the importance of genetic considerations during the treatment selection process. More research is needed in younger populations, given the lack of data on their safety profile.