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LEP

LEP is a leptin analog that activates leptin receptors to regulate appetite, energy expenditure, and metabolic homeostasis.

LEP is a leptin analog that activates leptin receptors to regulate appetite, energy expenditure, and metabolic homeostasis. Used for Congenital leptin deficiency, Lipodystrophy-associated metabolic complications.

At a glance

Mechanism of action

Leptin is an adipokine hormone that signals satiety and energy balance through the leptin receptor (OB-R) in the hypothalamus. LEP (metreleptin) is a recombinant human leptin replacement therapy that restores leptin signaling in patients with leptin deficiency or resistance, thereby reducing hyperphagia and promoting weight loss and metabolic normalization.

Approved indications

• Congenital leptin deficiency
• Lipodystrophy-associated metabolic complications

Common side effects

• Injection site reactions
• Headache
• Hypoglycemia
• Lymphoma (in animal studies)

Key clinical trials

• Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy (PHASE2)
• The Efficacy and Safety of the Combination of PD-1 With Chemotherapy and Adaptive Radiotherapy Strategy in the Treatment of Stage III Non-small Cell Lung Cancer Patients (PHASE2)
• Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy (PHASE3)
• Physical Exercise and Biomolecular Analysis to Reduce Uremic Toxins in Chronic Kidney Disease: An Exploratory Study (NA)
• Adipokines in Polycystic Ovary Syndrome
• The Influence of Non-Caloric Artificial Sweeteners on Youth With Type 1 Diabetes
• Expanded Access to REGN4461 for Patients With Diseases Associated With Deficient Leptin Signaling
• A Study Evaluating Mibavademab Treatment of Obesity Due to Leptin (LEP) Gene Mutations in Children, Adolescents and Adults (PHASE3)

Primary sources

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