Last reviewed 2026-04-20 · How we verify

Myalept (METRELEPTIN)

Myalept works by mimicking the action of leptin, a hormone that regulates fat distribution and metabolism.

At a glance

Mechanism of action

Adipocytes store lipids to meet the fuel requirements of non-adipose tissues during fasting. In patients with generalized lipodystrophy, the deficiency of adipose tissue leads to hypertriglyceridemia and ectopic deposition of fat in non-adipose tissues such as liver and muscle, contributing to metabolic abnormalities including insulin resistance. Native leptin is hormone predominantly secreted by adipose tissue that informs the central nervous system of the status of energy stores in the body. In patients with generalized lipodystrophy, leptin deficiency, resulting from the loss of adipose tissue, contributes to excess caloric intake, which exacerbates the metabolic abnormalities.MYALEPT (metreleptin) for injection exerts its function by binding to and activating the human leptin receptor (ObR), which belongs to the Class cytokine family of receptors that signals through the JAK/STAT transduction pathway.

Approved indications

• Acquired generalized lipodystrophy
• Acquired partial lipodystrophy
• Congenital generalized lipodystrophy

Boxed warnings

• 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 in patients with severe infections or loss of efficacy during MYALEPT treatment. Contact Amryt Pharmaceuticals DAC at 1-866-216-1526 for neutralizing antibody testing. ( 4.1 ), ( 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. ( 5.2 ) MYALEPT is available only through a restricted program called the MYALEPT REMS PROGRAM. ( 5.3 )

Common side effects

• Hypoglycemic events
• Headache
• Decreased weight
• Abdominal pain
• Arthralgia
• Dizziness
• Ear infection
• Fatigue
• Nausea
• Ovarian cyst
• Upper respiratory tract infection
• Anemia

Key clinical trials

• Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy (PHASE2)
• Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy (PHASE3)
• Natural History of Pregnancy and Pregnancy Outcomes in Metreleptin-Treated vs Untreated Subjects With Lipodystrophy
• Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy (PHASE3)
• Expanded Access Metreleptin Study
• MEASuRE: Metreleptin Effectiveness And Safety Registry
• Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy (PHASE3)
• Metreleptin in Anorexia Nervosa (PHASE2)

Patents

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Myalept CI brief — competitive landscape report
• Myalept updates RSS · CI watch RSS
• Amylin Pharms Llc portfolio CI