Subject: Myalept 1674-A SGM P2022

Drug

MYALEPT  (metreleptin)

Policy:

I.    INDICATIONS

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy. 

A.   Myalept is indicated as an adjunct to diet as replacement therapy to treat the complications of leptin
      deficiency in patients with congenital or acquired generalized lipodystrophy.

Limitations of Use:
1.   The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy
      have not been established.
2.   The safety and effectiveness of Myalept for the treatment of liver disease, including nonalcoholic
      steatohepatitis (NASH), have not been established.
3.   Myalept is not indicated for use in patients with HIV-related lipodystrophy.
4.   Myalept is not indicated for use in patients with metabolic disease, including diabetes mellitus and
      hypertriglyceridemia, without concurrent evidence of congenital or acquired generalized lipodystrophy.

B.   Compendial Use
      Partial lipodystrophy in patients with confirmed leptin deficiency and metabolic abnormalities 

All other indications are considered experimental/investigational and not medically necessary.

II.  DOCUMENTATION 

Submission of the following information is necessary to initiate the prior authorization review: leptin level (for initial requests)

III. EXCLUSIONS 

Coverage will not be provided for members with any of the following exclusions:
A.   HIV-related lipodystrophy
B.   Generalized obesity not associated with generalized lipodystrophy 

IV.  CRITERIA FOR INITIAL APPROVAL

Lipodystrophy
Authorization of 6 months may be granted for treatment of lipodystrophy when ALL of the following criteria are met:
A.   Member has a diagnosis of congenital generalized lipodystrophy (i.e., Berardinelli-Seip syndrome),
      acquired generalized lipodystrophy (i.e., Lawrence syndrome), or partial lipodystrophy
B.   Member has leptin deficiency confirmed by laboratory testing (i.e., less than 12 ng/ml)
C.   Member has at least one complication of lipodystrophy (e.g., diabetes mellitus, hypertriglyceridemia,
      increased fasting insulin level)

V.   CONTINUATION OF THERAPY

Lipodystrophy
Authorization of 12 months may be granted to members requesting continuation of treatment for lipodystrophy when the member has experienced an improvement from baseline in metabolic control (e.g., improved glycemic control, decrease in triglycerides, decrease in hepatic enzyme levels)

Place of Service:

Outpatient

The above policy is based on the following references:

1. Myalept [package insert]. Cambridge, MA: Aegerion Pharmaceuticals, Inc.; February 2022.  
2. Brown RJ, Araujo-Vilar D, Cheung PT, et al. The diagnosis and management of lipodystrophy syndromes: A multi-society practice guideline. J Clin Endocrinol Metab. 2016;101(12):4500-4511.
3. Handelsman Y, Oral AE, Bloomgarden ZT, et al. The clinical approach to the detection of lipodystrophy – an AACE consensus statement. Endocr Pract. 2013;19:107-116.
4. Chan JL, Lutz K, Cochran E, et al. Clinical effects of long-term metreleptin treatment in patients with lipodystrophy. Endocr Pract. 2011;17:922-932.
5. Garg A. Lipodystrophies: genetic and acquired body fat disorders. J Clin Endocrinol Metab. 2011;96:3313-3325.
6. Rodriguez AJ, Mastronardi CA, Paz-Filho GJ. New advances in the treatment of generalized lipodystrophy: role of metreleptin. Ther Clin Risk Manag. 2015; 11:1391-1400.
7. Lee HL, Waldman MA, Auh S, et al. Effects of metreleptin on proteinuria in patients with lipodystrophy. J Clin Endocrinol Metab.  2019 Apr 16. pii: jc.2019-00200. doi: 10.1210/jc.2019-00200. [Epub ahead of print].
8. Oral EA, Gorden P, Cochran E, et al. Long-term effectiveness and safety of metreleptin in the treatment of patients with partial lipodystrophy. Endocrine. 2019 Feb 25. doi: 10.1007/s12020-019-01862-8. [Epub ahead of print].

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

August 14, 2022