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Amifampridine Pharmacy Benefit Coverage Criteria Effective Date .......................................... 11/1/2020 Next Review Date… ................................... 11/1/2021 Coverage Policy Number ................................ P0074 Amifampridine Table of Contents Related Coverage Resources Medical Necessity Criteria ................................... 1 Quantity Limitations - (1201) FDA Approved Indications ................................... 2 Recommended Dosing ........................................ 2 Background .......................................................... 3 References .......................................................... 3 INSTRUCTIONS FOR USE The following Coverage Policy applies to health benefit plans administered by Cigna Companies. Certain Cigna Companies and/or lines of business only provide utilization review services to clients and do not make coverage determinations. References to standard benefit plan language and coverage determinations do not apply to those clients. Coverage Policies are intended to provide guidance in interpreting certain standard benefit plans administered by Cigna Companies. Please note, the terms of a customer’s particular benefit plan document [Group Service Agreement, Evidence of Coverage, Certificate of Coverage, Summary Plan Description (SPD) or similar plan document] may differ significantly from the standard benefit plans upon which these Coverage Policies are based. For example, a customer’s benefit plan document may contain a specific exclusion related to a topic addressed in a Coverage Policy. In the event of a conflict, a customer’s benefit plan document always supersedes the information in the Coverage Policies. In the absence of a controlling federal or state coverage mandate, benefits are ultimately determined by the terms of the applicable benefit plan document. Coverage determinations in each specific instance require consideration of 1) the terms of the applicable benefit plan document in effect on the date of service; 2) any applicable laws/regulations; 3) any relevant collateral source materials including Coverage Policies and; 4) the specific facts of the particular situation. Coverage Policies relate exclusively to the administration of health benefit plans. Coverage Policies are not recommendations for treatment and should never be used as treatment guidelines. In certain markets, delegated vendor guidelines may be used to support medical necessity and other coverage determinations. Medical Necessity Criteria Amifampridine (Firdapse® or Ruzurgi®) is considered medically necessary when ALL of the following criteria are met: • Individual is 6 years of age or older • Diagnosis of Lambert-Eaton myasthenic syndrome (LEMS) confirmed by ONE of the following: o Neurophysiology studies o Positive anti-P/Q type voltage – gated calcium Channel (VGCC) antibody testing • Attestation that the individual does not have a history of seizures • No concurrent use with other potassium channel blockers indicated for the treatment of LEMS • Prescribed by, or in consultation with, a neurologist Initial authorization is up to 3 months. Amifampridine (Firdapse® or Ruzurgi®) is considered medically necessary for continued use when the following criteria are met: • Individual continues to meet the initial criteria • Documentation of beneficial clinical response Reauthorization for up to 12 months. Page 1 of 4 Pharmacy Benefit Clinical Criteria: P0074 When coverage is available and medically necessary, the dosage, frequency, duration of therapy, and site of care should be reasonable, clinically appropriate, and supported by evidence-based literature and adjusted based upon severity, alternative available treatments, and previous response to therapy. Amifampridine (Firdapse® or Ruzurgi®) is considered experimental, investigational or unproven for ANY other use. Note: Receipt of sample product does not satisfy any criteria requirements for coverage. *If you’re a Cigna provider, please log in to the Cigna for Health Care Professionals website and search for specific patients to view their covered medications. FDA Approved Indications FDA Approved Indication Firdapse is a potassium channel blocker indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. Ruzurgi is a potassium channel blocker indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. Recommended Dosing FDA Recommended Dosing Firdapse Dosage Information: • The recommended starting dosage of FIRDAPSE is 15 mg to 30 mg daily, taken orally in divided doses (3 to 4 times daily). • The dosage can be increased by 5 mg daily every 3 or 4 days. • The maximum recommended total daily dosage is 80 mg. • The maximum single dose is 20 mg. • If a dose is missed, patients should not take double or extra doses. Ruzurgi Dosage Information: The recommended oral dosage for pediatric patients 6 to less than 17 years of age is dependent on body weight and is included in Table 1. Dosage should be increased based on clinical response and tolerability. If a dose is missed, patients should not take double or extra doses. Table 1: Recommended Dosage for Pediatric Patients 6 to Less Than 17 Years of Age Age and Body Initial Dosage Titration Regimen Maximum Maximum Weight Single Dose Total Daily Maintenance Dosage Pediatric patients 15 mg to 30 mg Increase daily in 5 30 mg 100 mg 6 to less than 17 daily, in divided mg to 10 mg years of age doses (2 to 3 times increments, divided weighing 45 kg per day) in up to 5 doses per or more day Pediatric patients 7.5 mg* to 15 mg Increase daily in 2.5 15 mg 50 mg 6 to less than 17 daily, in divided mg* to 5 mg years of age doses (2 to 3 times increments, divided weighing less per day) in up to 5 doses per than 45 kg day Page 2 of 4 Pharmacy Benefit Clinical Criteria: P0074 Drug Availability Firdapse and Ruzurgi are available as functionally scored oral tablets containing 10 mg amifampridine. Ruzurgi tablets may be used to prepare a 1mg/ml suspension for patients who require a dose less than 5mg. Background OVERVIEW Amifampridine is a broad spectrum potassium channel blocker.1,2 The mechanism by which amifampridine exerts its therapeutic effect in patients with Lambert-Eaton myasthenic syndrome (LEMS) has not been fully elucidated. • Firdapse is indicated for the treatment of LEMS in adults.1 • Ruzurgi is indicated for the treatment of LEMS in patients 6 years to < 17 years of age.2 Disease Overview LEMS is a rare autoimmune disorder affecting the connection between nerves and muscles and causing proximal muscle weakness, autonomic dysfunction, and areflexia.3,4 LEMS can occur at any age. The prevalence of LEMS specifically in pediatric patients is not known, but the overall prevalence of LEMS is estimated to be three per million individuals worldwide.3 The characteristic weakness is thought to be caused by antibodies generated against the P/Q-type voltage-gated calcium channels (VGCC) present on presynaptic nerve terminals and by diminished release of acetylcholine (ACh).4 More than half of LEMS cases are associated with small cell lung carcinoma (SCLC), which expresses functional VGCC. The diagnosis of LEMS is confirmed by electrodiagnostic studies, including repetitive nerve stimulation (RNS), or anti-P/Q-type VGCC antibody testing to confirm the diagnosis. Clinical Efficacy Firdapse was approved based on two pivotal trials.1,5 One pivotal trial enrolled both amifampridine-naïve and treatment-experienced patients; patients were initially entered into an open-label run-in phase lasting 90 days.5 During the open-label run-in phase, Firdapse was titrated for each individual patient to a dose that produced optimal neuromuscular benefit and tolerability in the opinion of the investigator. In order to continue in the study, treatment-naïve patients were required to have an improvement of at least three points in the quantitative myasthenia gravis score from the initial evaluation. Although Ruzurgi is indicated for use in children, the efficacy of Ruzurgi for the treatment of LEMS was established in one randomized, double-blind, placebo-controlled, withdrawal study in adults with an established diagnosis of LEMS (n = 32).2 Patients were required to be on an adequate and stable dosage for ≥ 3 months of Ruzurgi prior to entering the study. The efficacy of Ruzurgi in patients 6 to < 17 years of age is supported by evidence from adequate and well-controlled studies of Ruzurgi in adults with LEMS, pharmacokinetic data in adult patients, pharmacokinetic modeling and simulation to identify the dosing regimen in pediatric patients, and safety data from pediatric patients 6 to < 17 years of age. Safety Firdapse and Ruzurgi are contraindicated in patients with a history of seizures.1,2 There is also a Warning/Precaution in the prescribing information for these medications because seizures have been observed in patients with and without a history of seizures taking amifampridine at the recommended doses. Many of these patients were taking medications or had comorbidities that may have lowered their seizure threshold. Seizures may be dose-dependent. References 1. Firdapse® tablets [prescribing information]. Coral Gables, FL: Catalyst Pharmaceuticals, Inc.; November 2018. 2. Ruzurgi® tablets [prescribing information]. Princeton, NJ: Jacobus Pharmaceutical Company, Inc.; May 2019. Page 3 of 4 Pharmacy Benefit Clinical Criteria: P0074 3. FDA news release. FDA approves first treatment for children with Lambert-Eaton myasthenic syndrome,
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