MYALEPT (metreleptin)
MYALEPT (metreleptin) is a leptin analog indicated as an adjunct to diet for the treatment of complications arising from leptin deficiency. It is used as a replacement therapy specifically for patients with congenital or acquired generalized lipodystrophy. The drug is not intended for use in patients with partial lipodystrophy, HIV-related lipodystrophy, or metabolic diseases in the absence of generalized lipodystrophy. Its therapeutic role is to address the metabolic abnormalities, such as insulin resistance and hypertriglyceridemia, caused by the loss of adipose tissue.
How MYALEPT Works
MYALEPT functions by binding to and activating the human leptin receptor (ObR), which signals through the JAK/STAT transduction pathway. In patients with generalized lipodystrophy, a lack of adipose tissue leads to a deficiency of native leptin, a hormone that informs the central nervous system of the body's energy stores. By activating the leptin receptor, the drug helps regulate energy balance and reduces the excess caloric intake associated with the condition. This mechanism helps mitigate the ectopic deposition of fat in non-adipose tissues and the resulting metabolic complications.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2014-02-24
- Patent Cliff
- 2021
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
MYALEPT Approval History
What MYALEPT Treats
2 indicationsMYALEPT is approved for 2 conditions since its original approval in 2014. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Leptin Deficiency
- Generalized Lipodystrophy
MYALEPT Boxed Warning
RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with MYALEPT. The consequences of these neutralizing antibodies are not well characterized but could include inhibition of endogenous leptin action and/or loss of MYALEPT efficacy. Severe infection and/or worsening metabolic control have been reported. Test for anti-metreleptin antibodies with neutralizing activity in ...
WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with MYALEPT. The consequences of these neutralizing antibodies are not well characterized but could include inhibition of endogenous leptin action and/or loss of MYALEPT efficacy. Severe infection and/or worsening metabolic control have been reported. Test for anti-metreleptin antibodies with neutralizing activity in patients who develop severe infections or show signs suspicious for loss of MYALEPT efficacy during treatment. Contact Amryt Pharmaceuticals DAC at 1-866-216-1526 for neutralizing antibody testing of clinical samples [see Contraindications (4.1) and Warnings and Precautions (5.1) ]. T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with MYALEPT. Carefully consider the benefits and risks of treatment with MYALEPT in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy [see Warnings and Precautions (5.2) ]. Because of these risks associated with the development of anti-metreleptin antibodies that neutralize endogenous leptin and/or MYALEPT and the risk for lymphoma, MYALEPT is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the MYALEPT REMS PROGRAM [see Warnings and Precautions (5.3) ]. WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA See full prescribing information for complete boxed warning. Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with MYALEPT. The consequences are not well characterized but could include inhibition of endogenous leptin action and loss of MYALEPT efficacy. Worsening metabolic control and/or severe infection have been reported. Test for anti-metreleptin antibodies with neutralizing activity
MYALEPT Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Clinical Trial Registry
13 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT02262806 | 150002 15-DK-0002 | Ph 2 | active not recruiting | Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy |
| NCT02262832 | 150003 15-DK-0003 | Ph 3 | active not recruiting | Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy |
| NCT06679270 | APG-20-OLE APG-20 OLE | Ph 3 | recruiting | Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy |
| NCT06502990 | APL-20 2022-501781-22-00 | Ph 3 | recruiting | Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy |
| NCT06305182 METRAN | BASEC 2022-01328 | Ph 2 | recruiting | Metreleptin in Anorexia Nervosa |
| NCT04026178 results posted | AEGR-734-401 | Ph 4 | completed | Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy |
| NCT05164341 METRE-PL | APG-20 | Ph 3 | active not recruiting | Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL |
| NCT06484868 | APL-22 2022-502950-14-00 | Ph 4 | recruiting | Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy |
| NCT05351164 results posted | HUM00206598 | Ph 2 | active not recruiting | Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL) |
| NCT02654977 results posted | HUM00093399 | Ph 2 | completed | CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy |
| NCT01778556 results posted | 130057 13-DK-0057 | Ph 2 | completed | Short-term Effects of Leptin in People With Lipodystrophy |
| NCT01679197 results posted | MCRU 2834 R01DK088114-02 | Ph 2 | completed | Clinical Protocol to Investigate the Efficacy of Recombinant Human Leptin (Metreleptin) in Nonalcoholic Steatohepatitis (NASH) or Nonalcoholic Fatty Liver Disease (NAFLD) Associated With Lipodystrophy |
| NCT00673387 results posted | DFA102 | Ph 2 | completed | Study to Examine Safety, Tolerability, and Effect on Body Weight of Metreleptin Administered in Conjunction With Pramlintide in Obese and Overweight Subjects |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
MYALEPT FDA Label Details
Indications & Usage
FDA Label (PDF)MYALEPT is indicated for the treatment of Leptin Deficiency; Generalized Lipodystrophy.
WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with MYALEPT. The consequences of these neutralizing antibodies are not well characterized but could include in...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment