Currentmedical mutual - ohioPolicy N/A

Myalept (metreleptin) Prior Authorization Policy

Prior authorization guidance for pharmacy benefit coverage of Myalept (metreleptin) for treatment of complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy, including initial and continuation criteria, duration of approvals, conditions not recommended for approval, and documentation requirements.

Policy Summary

Payermedical mutual - ohio

PolicyMyalept (metreleptin) Prior Authorization Policy

Policy CodePolicy N/A

Change TypeAnnual review — no material changes

Effective Date

Next Review DateSep 18, 2025

Key ActionPrior authorization is recommended for pharmacy benefit coverage of Myalept; approval recommended when member meets the criteria in the policy.

POLICY UPDATE CHANGES

No material clinical/coverage changes identified on this review (has_material_change=false).

1FDA-Approved Indication (generalized lipodystrophy)

5Conditions Not Recommended (examples listed)

1 yrInitial approval duration

Coverage Summary

Myalept (metreleptin) coverage stance: covered with criteria for the FDA‑approved indication of generalized lipodystrophy (congenital or acquired). Scope: prior authorization guidance for pharmacy benefit coverage including initial and continuation criteria, 1‑year initial and 1‑year extended approval durations, requirements for specialist prescribing, and documentation requirements. Safety/REMS: Myalept carries boxed warnings for risk of lymphoma and for development of neutralizing anti‑metreleptin antibodies; it is available only through a REMS program that requires practitioner training and use of a REMS Prescription Authorization Form.

Initial Therapy Criteria (FDA-Approved Indication)

FDA-Approved Indication: Generalized Lipodystrophy (Congenital or Acquired)

Approve for duration noted if the patient meets ALL of the following criteria (A, B, C, D, and E):

ALL of the following

A) Patient meets ONE of the following (i or ii)
- i. Congenital generalized lipodystrophy - ONE of (a or b)
  - a) Genetic test demonstrating one gene mutation confirming diagnosis: a) Patient has had a genetic test demonstrating one gene mutation (i.e., AGPAT2, BSCL2, CAV1, or PTRF) confirming the diagnosis of congenital generalized lipodystrophy
  - b) BOTH of the following (1 and 2)
    (1) Patient has had a genetic test that did not demonstrate an AGPAT2, BSCL2, CAV1, or PTRF gene mutation
    (2) A clinical diagnosis of congenital generalized lipodystrophy has been made by a specialist with experience in treating patients with lipodystrophy
- ii. Acquired generalized lipodystrophy: ii. Patient has acquired generalized lipodystrophy
B) Manifestations of leptin deficiency: B) Patient has experienced one or more manifestations of leptin deficiency (note: manifestations include hyperinsulinemia, type 2 diabetes mellitus, hypertriglyceridemia)
C) Conjunction with dietary modification: C) Myalept will be used in conjunction with dietary modification
D) Failure of conventional therapy: D) Conventional therapy for metabolic disturbances has failed (e.g., diet and lifestyle modification, statins, anti-diabetic agents)
E) Specialist prescriber requirement: E) Medication is prescribed by or in consultation with an endocrinologist or a geneticist

Continuation Therapy Criteria

Continuation of Therapy

Approve for continuation if ALL of the following are met:

ALL of the following

Patient meets all criteria in the initial FDA-Approved Indication section
Patient's condition has improved or stabilized while using Myalept (evidenced by sustained improvement in triglyceride levels, hemoglobin A1c from baseline, or provider confirmation that Myalept is still working)

Conditions Not Recommended for Approval

Myalept is not recommended for approval for the following conditions (not exhaustive):

ANY of the following

General obesity not associated with congenital leptin deficiency (Myalept contraindicated)
Liver disease, including nonalcoholic steatohepatitis (NASH)
Partial lipodystrophy (safety and efficacy not established; prescribing information lists as limitation of use)
HIV-related lipodystrophy (not indicated)
Metabolic disease without generalized lipodystrophy: Metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy
Other circumstances: Circumstances not listed in the Recommended Authorization Criteria

Provider Actions & Authorization Requirements

Prior Authorization

Prior authorization required

Prior authorization is recommended for pharmacy benefit coverage of Myalept; approval recommended when member meets the criteria in the policy.

Documentation Required

Documentation to support medical necessity

The Company may request additional documentation including patient records, test results, and provider credentials; claims may be denied if the drug is not medically necessary, investigational, experimental, or outside the member's benefits.

Documentation Required

Specialist prescribing requirement for initial therapy

Initial approval requires Myalept be prescribed by or in consultation with an endocrinologist or a geneticist.

Documentation Required

REMS program and training

Prescribers must complete REMS training and utilize a Myalept REMS Prescription Authorization Form for each new Myalept prescription due to boxed warnings and REMS requirements.

Background and Definitions

Myalept is a recombinant analog of human leptin indicated as an adjunct to diet to treat complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. The drug replaces leptin to address metabolic complications arising from inadequate adipose tissue. Important safety information: Myalept has two Boxed Warnings—one for the risk of lymphoma and one for the development of neutralizing anti‑metreleptin antibodies that may cause loss of endogenous leptin activity or loss of Myalept efficacy. Because of these serious risks, Myalept is available only through a REMS program, which requires prescriber training and completion of a Myalept REMS Prescription Authorization Form for each new prescription.

Manifestations of leptin deficiency include: hyperinsulinemia, type 2 diabetes mellitus, and hypertriglyceridemia.

Policy Summary

Payermedical mutual - ohio

PolicyMyalept (metreleptin) Prior Authorization Policy

Policy CodePolicy N/A

Change TypeAnnual review — no material changes

Effective Date

Next Review DateSep 18, 2025

Key ActionPrior authorization is recommended for pharmacy benefit coverage of Myalept; approval recommended when member meets the criteria in the policy.

On This Page

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Myalept (metreleptin) Prior Authorization Policy

Coverage Summary

Initial Therapy Criteria (FDA-Approved Indication)

Continuation Therapy Criteria

Initial and Extended Approval Duration

Conditions Not Recommended for Approval

Provider Actions & Authorization Requirements

Applicable Codes

Background and Definitions

Clinical Evidence

Revision History