MYALEPT
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.
Details
- Status
- Prescription
- First Approved
- 2014-02-24
- 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
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
ProIndications & Usage
FDA Label (PDF)MYALEPT is a leptin analog 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 The safety and effectiveness of MYALEPT for the treatment of complications of partial lipodystrophy have not been established. The safety and effectiveness of MYALEPT for the treatment of liver disease, including nonalcoholic steatohepatitis (NASH), have not been established. MYALEPT is not indicated for use in patients with HIV-related lipodystrophy. MYALEPT is not indicated for u...
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.