Summary

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• Firdapse (amifampridine) is indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and pediatric patients 6 years of age and older.
• This summary is based on the review of four systematic review(s)/meta-analysis(es). ^[1-4]
• Treatment with 3,4-diaminopyridine (3,4-DAP) resulted in a significant improvement in Quantitative Myasthenia Gravis (QMG) score by 2.76 points (95% CI, -4.08 to -1.45, p < 0.001). Additionally, two phase III trials for amifampridine showed significant improvements in QMG scores at days 4 and 14 compared to placebo. 4. (3,4-DAP) significantly improved Compound Muscle Action Potentials (CMAP) amplitude compared to placebo, with a mean difference of 1.34 mV (95% CI, 0.98 to 1.70, p < 0.001).
• Amifampridine demonstrated a significant improvement in Subjective Global Impression scores, indicating enhanced patient-perceived symptom relief.
• General Safety: Amifampridine and related compounds (3,4-DAP and 3,4-DAPP) are generally well-tolerated, with common adverse effects including peri-oral and finger paresthesia and gastrointestinal side effects, which are considered tolerable by patients. Serious side reactions, such as seizures, are not a concern if the daily dose remains under 80 mg.
• Long-term Use: AFPs, including amifampridine, are proven to be safe for long-term use, and their safety profile is enhanced by supplementing with pyridostigmine to maintain symptom control without undue side effects.
• There is no population types or subgroups information available in the reviewed studies.