Drug updated on 9/5/2024
Dosage Form | Tablet (oral; 10 mg) |
Drug Class | Potassium channel blockers |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and pediatric patients 6 years of age and older.
Latest News
Summary
- 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.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Firdapse (amifampridine) Prescribing Information. | 2023 | Catalyst Pharmaceuticals, Coral Gables, FL |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
3,4-diaminopyridine treatment for Lambert-Eaton myasthenic syndrome in adults: a meta-analysis of randomized controlled trials. | 2021 | BMC Neurology |
Amifampridine for the management of lambert-eaton myasthenic syndrome: a new take on an old drug. | 2020 | Annals of Pharmacotherapy |
Amifampridine for the treatment of Lambert-Eaton myasthenic syndrome. | 2019 | Expert Review of Clinical Immunology |
Amifampridine tablets for the treatment of Lambert-Eaton myasthenic syndrome. | 2019 | Expert Review of Clinical Immunology |
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Guideline for the management of myasthenic syndromes. | 2023 | Therapeutic Advances in Neurological Disorders |
Update in the management of myasthenia gravis and lambert-eaton myasthenic syndrome. | 2021 | Neurologic Clinics |