Summary

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• Cerdelga (eliglustat) is indicated for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.
• This summary is based on the review of one systematic review/meta-analysis. ^[1]
• Eliglustat (Cerdelga): In experienced patients with Gaucher disease type 1, eliglustat maintained stable outcomes, indicating effectiveness in maintaining patient condition over time.
• Enzyme Replacement Therapy (ERT): ERTs, including imiglucerase, velaglucerase alfa, and taliglucerase alfa, were effective in both naïve and experienced patients with Gaucher disease type 1, with confirmed effectiveness in both short- and long-term follow-ups.
• Miglustat (SRT): Miglustat did not significantly improve blood outcomes in naïve patients and was associated with decreased platelet levels in experienced patients, indicating less favorable effectiveness compared to eliglustat and ERTs.
• There is no safety information available in the reviewed studies regarding eliglustat.
• Miglustat showed a decrease in platelet levels in experienced patients, indicating a potential safety concern.
• The systematic review focused on adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs). Eliglustat showed stable outcomes in experienced patients; no detailed subgroup analyses by other demographic factors or comorbid conditions were provided.