The Amifampridine Phosphate 
Expanded Access Program (EAP)

The Amifampridine Phosphate 
Expanded Access Program (EAP)

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The investigational treatment amifampridine phosphate may be available at no cost to patients diagnosed with the neuromuscular disorders Lambert-Eaton Myasthenic Syndrome (LEMS) or certain types of Congenital Myasthenic Syndromes (CMS)

Expanded Access Programs are intended to give patients access to investigational medications for serious diseases or conditions when there is no comparable or satisfactory treatment available. Participating physicians are able to provide the medication to their eligible U.S. patients while it is still under development.

The FDA has not yet approved amifampridine phosphate for the treatment of either LEMS or CMS.

WHAT IS LEMS?

Lambert-Eaton Myasthenic Syndrome, or LEMS, is a rare autoimmune disorder that causes muscle weakness and affects muscle function.1-5 In LEMS, your body attacks nerve endings that regulate a neurotransmitter controlling muscle contractions.2,4 LEMS affects approximately 1 per 100,000 persons.About 50%-60% of LEMS patients have an underlying cancer–– most commonly small cell lung cancer.2,3

WHAT ARE THE SYMPTOMS 
OF LEMS?

Common symptoms include muscle weakness generally starting in the upper legs and hips, as well as fatigue, muscle pain and stiffness.2,6,7 These symptoms may affect your activities of daily living; for example, you might have difficulty walking, getting out of a chair or climbing stairs or you could fall more easily.  Other signs of LEMS may include reduced reflexes, drooping eyelids, facial weakness, and problems swallowing.2,7

HOW IS LEMS DIAGNOSED?

LEMS is typically diagnosed by a neurologist specializing in neuromuscular disorders. The diagnosis is based on the results of tests that measure electrical activity in muscles (electromyography, or EMG), and can be confirmed by blood tests for specific antibodies.

WHAT IS CMS?

Congenital Myasthenic Syndromes (CMS), are a group of rare, inherited diseases that primarily cause muscle weakness.8 CMS is caused by problems with genes that affect communication between nerve and muscle cells.  Defects in at least 20 different genes have been associated with CMS.8

Because CMS is frequently misdiagnosed or undiagnosed, there is no reliable information on how many people are affected. Best estimates are that CMS affects approximately 3 per 1 million individuals in the United States.

WHAT ARE THE SYMPTOMS 
OF CMS?

Typically patients with CMS exhibit muscle weakness, fatigue, and droopy eyelids but symptoms may vary depending on the type of CMS. Symptoms often appear in early childhood.8-10

HOW IS CMS DIAGNOSED?

CMS is diagnosed by a neurologist specializing in neuromuscular disorders. The physician typically begins by reviewing a patient’s medical history and conducting a physical exam to determine the extent of muscle weakness. The diagnosis may include blood tests, tests to measure electrical activity of muscles (electromyography, or EMG), and genetic testing. Genetic testing will be covered through the EAP if it has not previously been done and is not covered by the patient’s insurance.

WHAT IS AN EXPANDED ACCESS PROGRAM (EAP)?

Expanded Access Programs are intended to give patients access to investigational medications for serious diseases or conditions when there is no comparable or satisfactory treatment available. Investigational medications are authorized for use through an Expanded Access Program by the FDA. Participating physicians are then able to obtain and provide the medication to their eligible U.S. patients while the medication is still under development.

ABOUT THE AMIFAMPRIDINE PHOSPHATE EXPANDED ACCESS PROGRAM (EAP)

The Expanded Access Program is designed to provide access to the investigational treatment amifampridine phosphate for patients diagnosed with LEMS or certain types of CMS.  At this time, amifampridine phosphate is only available through the EAP when a patient’s treating physician feels this treatment can help improve their condition.  Please discuss your treatment options with your physician.

Catalyst Pharmaceuticals is committed to ensuring all eligible patients have access to treatment. The FDA has not yet approved amifampridine phosphate for the symptomatic treatment of either LEMS or CMS.

ABOUT AMIFAMPRIDINE PHOSPHATE

Amifampridine phosphate is an investigational drug that has shown positive results in the treatment of LEMS in a recent U.S. clinical study.  No serious side effects were reported.  The most common side effects were: digital and oral paresthesias (e.g., tingling of the fingers, toes, or area around the mouth); nausea; and headache. Amifampridine phosphate may be available at no cost for patients diagnosed with Lambert-Eaton Myasthenic Syndrome (LEMS) or certain types of Congenital Myasthenic Syndromes (CMS) through this Expanded Access Program (EAP).

Amifampridine phosphate has been approved and marketed for more than 5 years in the European Union for the treatment of LEMS. The FDA has not yet approved amifampridine phosphate for the symptomatic treatment of either LEMS or CMS.

Catalyst Pharmaceuticals is committed to ensuring all eligible patients access to treatment.

Please visit the Global Genes website, Living with LEMS, for additional information about LEMS and support for LEMS patients.

References: 1. Sanders DB. Lambert-Eaton myasthenic syndrome: diagnosis and treatment. Ann N Y Acad Sci. 2003;998:500-558. 2. Titulaer MJ, Lang B, Verschuuren JJ. Lambert-Eaton myasthenic syndrome: from clinical characteristics to therapeutic strategies. Lancet Neurol. 2011;10(12):1098-1107. 3. Titulaer MJ, Maddison P, Sont JK, et al. Clinical Dutch-English Lambert-Eaton Myasthenic Syndrome (LEMS) Tumor Association Prediction score accurately predicts small-cell lung cancer in the LEMS. J Clin Oncol. 2011;29(7): 902-908. 4. Oh SJ, Sieb JP. Update on amifampridine as a drug of choice in Lambert-Eaton myathenic syndrome. US Neurol. 2014;10(2):83-89. 5. Oh SJ, Kurokawa K, Claussen GC, Ryan HF Jr. Electrophysiological diagnostic criteria of Lambert-Eaton myasthenic syndrome. Muscle Nerve. 2005;32(4):515-520. 6. Harms l, Sieb JP, Williams AE et al. Long-term disease history, clinical symptoms,health status, and healthcare utilization in patients suffering from Lambert Eatonmyasthenic syndrome: Results of a patient interview survey in GermanyJournal of Medical Economics Vol. 15, No.3, 2012,1-10 7. Stickler D. Lambert-Eaton Myasthenic Syndromes (LEMS) Clinical Presentation. Medscape. http://emedicine.medscape.com/article/1170810-clinical. Accessed March 7, 2016. 8. Engel AG, Shen XM, Selcen D, Sine SM. Congenital myasthenic syndromes: pathogenesis, diagnosis, and treatment. Lancet Neurol. 2015;14(4):420-434. 9. Prevalence of rare diseases: bibliographic data. Orphanet Report Series, Rare Diseases collection. July 2015; number 2. http://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_ decreasing_prevalence_or_cases.pdf. Accessed September 22, 2015. 10. Eymard B, Hantaï D, Estournet B. Congenital myasthenic syndromes. Handb Clin Neurol. 2013;113:1469-1480.

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THE AMIFAMPRIDINE PHOSPHATE EAP, LEMS AND CMS