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LEMS EDUCATIONAL VIDEO LIBRARY

Why LEMS Warrants a Closer Look: Clinical Insights and Common Misdiagnoses

This educational video series describes the clinical presentation of Lambert-Eaton myasthenic syndrome (LEMS), the challenges of diagnosis, and the role that FIRDAPSE® can play in treatment. This content was featured in a presentation delivered at the 2019 Meeting of the American Association of Neuromuscular & Electrodiagnostic Medicine.

CHAPTER 1—LEMS PATHOPHYSIOLOGY AND CLINICAL PRESENTATION

This video describes disorders of the neuromuscular junction, mechanisms of disease, and the pathophysiology, classification, and clinical presentation of LEMS.

Presented by Perry B. Shieh, MD, PhD
Department of Neurology
David Geffen School of Medicine at UCLA

CHAPTER 2—DIAGNOSIS AND MISDIAGNOSIS IN LEMS

This video presents diagnostic approaches for LEMS, common misdiagnoses, and the expected progression of disease.

Presented by Perry B. Shieh, MD, PhD
Department of Neurology
David Geffen School of Medicine at UCLA

CHAPTER 3—THE ROLE OF FIRDAPSE IN LEMS TREATMENT

This video provides a clinical overview of FIRDAPSE, including its MOA and the efficacy and safety results from two Phase 3 clinical trials.

Presented by Perry B. Shieh, MD, PhD
Department of Neurology
David Geffen School of Medicine at UCLA

This program and its materials have been sponsored by Catalyst Pharmaceuticals. The speaker has been compensated by Catalyst Pharmaceuticals to provide this presentation. This presentation is consistent with all applicable US Food and Drug Administration (FDA) requirements and the approved product labeling for FIRDAPSE. This program is not intended for continuing education credit for healthcare professionals. Any expenditures in relation to this event may be reported as required by state and federal spend-reporting requirements.

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Indication and Important Safety Information

indications and usage:

FIRDAPSE is a potassium channel blocker indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and pediatric patients 6 years of age and older.

CONTRAINDICATIONS

FIRDAPSE is contraindicated in patients with:

  • A history of seizures
  • Hypersensitivity to amifampridine phosphate or another aminopyridine

WARNINGS AND PRECAUTIONS

Seizures: FIRDAPSE can cause seizures. Consider discontinuation or dose-reduction of FIRDAPSE in patients who have a seizure while on treatment.

Hypersensitivity: If a hypersensitivity reaction such as anaphylaxis occurs, FIRDAPSE should be discontinued and appropriate therapy initiated.

ADVERSE REACTIONS

The most common (> 10%) adverse reactions are: paresthesia, upper respiratory tract infection, abdominal pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension, and muscle spasms.

Please see full Prescribing Information for additional Important Safety Information.

To report SUSPECTED ADVERSE REACTIONS, contact Catalyst Pharmaceuticals at 1-844-347-3277 (1-844-FIRDAPSE) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

References:
  1. Yoon CH, Owusu-Guha J, Smith A, Buschur P. Amifampridine for the management of Lambert-Eaton myasthenic syndrome: a new take on an old drug. Ann Pharmacother. 2020;54(1):56-63.
  2. Full Prescribing Information for FIRDAPSE (amifampridine). Catalyst Pharma; 2022.
  3. Shieh P, Sharma K, Korhman B, Oh SJ. Amifampridine phosphate (FIRDAPSE) is effective in a confirmatory phase 3 clinical trial in LEMS. J Clin Neuromuscul Dis. 2019;20(3):111-119.
  4. Oh SJ, Scherbakova N, Kostera-Pruszczyk A, et al: LEMS Study Group. Amifampridine phosphate (FIRDAPSE®) is effective and safe in a phase 3 clinical trial in LEMS. Muscle Nerve. 2016;53(5):717-725.
  5. Lennon VA, Kryzer TJ, Griesmann GE, et al. Calcium-channel antibodies in the Lambert-Eaton syndrome and other paraneoplastic syndromes. N Engl J Med. 1995;332(22):1467-1474.
  6. Skeie GO, Apostolski S, Evoli A, et al. Guidelines for treatment of autoimmune neuromuscular transmission disorders. Eur J Neurol. 2010;17(7):893-902.
  7. Data on file, Catalyst Pharmaceuticals.
  8. Orange Book: Approved drug products with therapeutic equivalence evaluations. US Food and Drug Administration website. https://www.accessdata.fda.gov/scripts/cder/ob/search_product.cfm. Accessed June 10, 2020.
  9. Muppidi S, Wolfe GI, Barohn RJ. Diseases of the neuromuscular junction. In: Swaiman K, Ashwal S, Ferriero D, Schor N, eds. Pediatric Neurology: Principles and Practice. 5th ed. Philadelphia, PA: Elsevier; 2011:1549-1569.
  10. Sanders DB. Lambert-Eaton myasthenic syndrome: diagnosis and treatment. Ann NY Acad Sci. 2003;998:500-508.
  11. Harms L, Sieb JP, Williams AE, et al. Long-term disease history, clinical symptoms, health status, and healthcare utilization in patients suffering from Lambert Eaton myasthenic syndrome: results of a patient interview survey in Germany. J Med Econ. 2012;15(3):521-530.
  12. Merino-Ramírez MÁ, Bolton CF. Review of the diagnostic challenges of Lambert-Eaton syndrome revealed through three case reports. Can J Neurol Sci. 2016;43(5):635-647.
  13. Titulaer MJ, Lang B, Verschuuren JJ. Lambert-Eaton myasthenic syndrome: from clinical characteristics to therapeutic strategies. Lancet Neurol. 2011;10(12):1098-1107.
  14. Titulaer MJ, Wirtz PW, Willems LNA, et al. Screening for small-cell lung cancer: a follow-up study of patients with Lambert-Eaton myasthenic syndrome. J Clin Oncol. 2008;26(26):4276-4281.
  15. Wirtz PW, Smallegange TM, Wintzen AR, Verschuuren JJ. Differences in clinical features between the Lambert-Eaton myasthenic syndrome with and without cancer: an analysis of 227 published cases. Clin Neurol Neurosurg. 2002;104(4):359-363.
  16. Wirtz PW, Wintzen AR, Verschuuren JJ. Lambert-Eaton myasthenic syndrome has a more progressive course in patients with lung cancer. Muscle Nerve. 2005;32(2):226-229.
  17. Maddison P, Lang B, Mill K, Newsom-Davis J. Long term outcome in Lambert-Eaton myasthenic syndrome without lung cancer. J Neurol Neurosurg Psychiatry. 2001;70(2):212-217.
  18. Zalewski NL, Lennon VA, Lachance DH, et al. P/Q- and N-type calcium-channel antibodies: oncological, neurological, and serological accompaniments. Muscle Nerve. 2016;54(2):220-227.
  19. Lennon VA. Serologic profile of myasthenia gravis and distinction from the Lambert-Eaton myasthenic syndrome. Neurology. 1997;48(suppl 5):S23-S27.
  20. Gilhus NE. Lambert-Eaton myasthenic syndrome; pathogenesis, diagnosis, and therapy. Autoimmune Dis. 2011;2011:973808.
  21. Quartel A, Turbeville S, Lounsbury D. Current therapy for Lambert-Eaton myasthenic syndrome: development of 3,4-diaminopyridine phosphate salt as first-line symptomatic treatment. Curr Med Res Opin. 2010;26(6):1363-1375.
  22. Chalk CH, Murray NM, Newsom-Davis J, O'Neill JH, Spiro SG. Response of the Lambert-Eaton myasthenic syndrome to treatment of associated small-cell lung carcinoma. Neurology. 1990;40(10):1552-1556.
  23. Hyun JW, Kim SH, Jeong IH, Kim HT, Kim HJ. Successful treatment of Lambert-Eaton myasthenic syndrome in a small cell lung cancer patient using 3,4-diaminopyridine: a case report. Onc Ltrs. 2016;11:1933-1935.
  24. Bain PG, Motomura M, Newsom-Davis J, et al. Effects of intravenous immunoglobulin on muscle weakness and calcium-channel autoantibodies in the Lambert-Eaton myasthenic syndrome. Neurology. 1996;47(3):678-683.
  25. Ivanovski T, Miralles F. Lambert-Eaton myasthenic syndrome: early diagnosis is key. Degener Neurol Neuromuscul Dis. 2019;9:27-37.
  26. Wirtz PW, Verschuuren JJ, van Dijk JG, et al. Efficacy of 3,4-diaminopyridine and pyridostigmine in the treatment of Lambert-Eaton myasthenic syndrome: a randomized, double-blind, placebo-controlled, crossover study. Clin Pharmacol Ther. 2009;86(1):44-48.
  27. Oh SJ, Sieb JP. Update on amifampridine as a drug of choice in Lambert-Eaton myasthenic syndrome. US J Neurol. 2014;10(2):83-89.
  28. Strupp M, Teufel J, Zwergal A, et al. Aminopyridines for the treatment of neurologic disorders. Neurol Clin Pract. 2017;7(1):65-76.
  29. Lindquist S, Stangel M. Update on treatment options for Lambert-Eaton myasthenic syndrome: focus on use of amifampridine. Neuropsychiatr Dis Treat. 2011;7:341-349.
  30. Jacob S, Muppidi S, Guidon A, et al; International MG/COVID-19 Working Group. Guidance for the management of myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) during the COVID-19 pandemic. J Neurol Sci. 2020;412:116803.

Indication and Important Safety Information

indications and usage:

FIRDAPSE is a potassium channel blocker indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults and pediatric patients 6 years of age and older.

CONTRAINDICATIONS

FIRDAPSE is contraindicated in patients with:

  • A history of seizures
  • Hypersensitivity to amifampridine phosphate or another aminopyridine

WARNINGS AND PRECAUTIONS

Seizures: FIRDAPSE can cause seizures. Consider discontinuation or dose-reduction of FIRDAPSE in patients who have a seizure while on treatment.

Hypersensitivity: If a hypersensitivity reaction such as anaphylaxis occurs, FIRDAPSE should be discontinued and appropriate therapy initiated.

ADVERSE REACTIONS

The most common (> 10%) adverse reactions are: paresthesia, upper respiratory tract infection, abdominal pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension, and muscle spasms.

Please see full Prescribing Information for additional Important Safety Information.

To report SUSPECTED ADVERSE REACTIONS, contact Catalyst Pharmaceuticals at 1-844-347-3277 (1-844-FIRDAPSE) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

References:
  1. Yoon CH, Owusu-Guha J, Smith A, Buschur P. Amifampridine for the management of Lambert-Eaton myasthenic syndrome: a new take on an old drug. Ann Pharmacother. 2020;54(1):56-63.
  2. Full Prescribing Information for FIRDAPSE (amifampridine). Catalyst Pharma; 2022.
  3. Shieh P, Sharma K, Korhman B, Oh SJ. Amifampridine phosphate (FIRDAPSE) is effective in a confirmatory phase 3 clinical trial in LEMS. J Clin Neuromuscul Dis. 2019;20(3):111-119.
  4. Oh SJ, Scherbakova N, Kostera-Pruszczyk A, et al: LEMS Study Group. Amifampridine phosphate (FIRDAPSE®) is effective and safe in a phase 3 clinical trial in LEMS. Muscle Nerve. 2016;53(5):717-725.
  5. Lennon VA, Kryzer TJ, Griesmann GE, et al. Calcium-channel antibodies in the Lambert-Eaton syndrome and other paraneoplastic syndromes. N Engl J Med. 1995;332(22):1467-1474.
  6. Skeie GO, Apostolski S, Evoli A, et al. Guidelines for treatment of autoimmune neuromuscular transmission disorders. Eur J Neurol. 2010;17(7):893-902.
  7. Data on file, Catalyst Pharmaceuticals.
  8. Orange Book: Approved drug products with therapeutic equivalence evaluations. US Food and Drug Administration website. https://www.accessdata.fda.gov/scripts/cder/ob/search_product.cfm. Accessed June 10, 2020.
  9. Muppidi S, Wolfe GI, Barohn RJ. Diseases of the neuromuscular junction. In: Swaiman K, Ashwal S, Ferriero D, Schor N, eds. Pediatric Neurology: Principles and Practice. 5th ed. Philadelphia, PA: Elsevier; 2011:1549-1569.
  10. Sanders DB. Lambert-Eaton myasthenic syndrome: diagnosis and treatment. Ann NY Acad Sci. 2003;998:500-508.
  11. Harms L, Sieb JP, Williams AE, et al. Long-term disease history, clinical symptoms, health status, and healthcare utilization in patients suffering from Lambert Eaton myasthenic syndrome: results of a patient interview survey in Germany. J Med Econ. 2012;15(3):521-530.
  12. Merino-Ramírez MÁ, Bolton CF. Review of the diagnostic challenges of Lambert-Eaton syndrome revealed through three case reports. Can J Neurol Sci. 2016;43(5):635-647.
  13. Titulaer MJ, Lang B, Verschuuren JJ. Lambert-Eaton myasthenic syndrome: from clinical characteristics to therapeutic strategies. Lancet Neurol. 2011;10(12):1098-1107.
  14. Titulaer MJ, Wirtz PW, Willems LNA, et al. Screening for small-cell lung cancer: a follow-up study of patients with Lambert-Eaton myasthenic syndrome. J Clin Oncol. 2008;26(26):4276-4281.
  15. Wirtz PW, Smallegange TM, Wintzen AR, Verschuuren JJ. Differences in clinical features between the Lambert-Eaton myasthenic syndrome with and without cancer: an analysis of 227 published cases. Clin Neurol Neurosurg. 2002;104(4):359-363.
  16. Wirtz PW, Wintzen AR, Verschuuren JJ. Lambert-Eaton myasthenic syndrome has a more progressive course in patients with lung cancer. Muscle Nerve. 2005;32(2):226-229.
  17. Maddison P, Lang B, Mill K, Newsom-Davis J. Long term outcome in Lambert-Eaton myasthenic syndrome without lung cancer. J Neurol Neurosurg Psychiatry. 2001;70(2):212-217.
  18. Zalewski NL, Lennon VA, Lachance DH, et al. P/Q- and N-type calcium-channel antibodies: oncological, neurological, and serological accompaniments. Muscle Nerve. 2016;54(2):220-227.
  19. Lennon VA. Serologic profile of myasthenia gravis and distinction from the Lambert-Eaton myasthenic syndrome. Neurology. 1997;48(suppl 5):S23-S27.
  20. Gilhus NE. Lambert-Eaton myasthenic syndrome; pathogenesis, diagnosis, and therapy. Autoimmune Dis. 2011;2011:973808.
  21. Quartel A, Turbeville S, Lounsbury D. Current therapy for Lambert-Eaton myasthenic syndrome: development of 3,4-diaminopyridine phosphate salt as first-line symptomatic treatment. Curr Med Res Opin. 2010;26(6):1363-1375.
  22. Chalk CH, Murray NM, Newsom-Davis J, O'Neill JH, Spiro SG. Response of the Lambert-Eaton myasthenic syndrome to treatment of associated small-cell lung carcinoma. Neurology. 1990;40(10):1552-1556.
  23. Hyun JW, Kim SH, Jeong IH, Kim HT, Kim HJ. Successful treatment of Lambert-Eaton myasthenic syndrome in a small cell lung cancer patient using 3,4-diaminopyridine: a case report. Onc Ltrs. 2016;11:1933-1935.
  24. Bain PG, Motomura M, Newsom-Davis J, et al. Effects of intravenous immunoglobulin on muscle weakness and calcium-channel autoantibodies in the Lambert-Eaton myasthenic syndrome. Neurology. 1996;47(3):678-683.
  25. Ivanovski T, Miralles F. Lambert-Eaton myasthenic syndrome: early diagnosis is key. Degener Neurol Neuromuscul Dis. 2019;9:27-37.
  26. Wirtz PW, Verschuuren JJ, van Dijk JG, et al. Efficacy of 3,4-diaminopyridine and pyridostigmine in the treatment of Lambert-Eaton myasthenic syndrome: a randomized, double-blind, placebo-controlled, crossover study. Clin Pharmacol Ther. 2009;86(1):44-48.
  27. Oh SJ, Sieb JP. Update on amifampridine as a drug of choice in Lambert-Eaton myasthenic syndrome. US J Neurol. 2014;10(2):83-89.
  28. Strupp M, Teufel J, Zwergal A, et al. Aminopyridines for the treatment of neurologic disorders. Neurol Clin Pract. 2017;7(1):65-76.
  29. Lindquist S, Stangel M. Update on treatment options for Lambert-Eaton myasthenic syndrome: focus on use of amifampridine. Neuropsychiatr Dis Treat. 2011;7:341-349.
  30. Jacob S, Muppidi S, Guidon A, et al; International MG/COVID-19 Working Group. Guidance for the management of myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS) during the COVID-19 pandemic. J Neurol Sci. 2020;412:116803.