Leptin
Also known as: LEP, Metreleptin, Myalept, Myalepta, Ob gene product, OB protein, rmetHuLeptin
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Summary
Leptin is a 167‑amino‑acid peptide hormone secreted by adipocytes that signals nutritional status to the hypothalamus. Recombinant human leptin (metreleptin) is used clinically for congenital leptin deficiency and generalized lipodystrophy.
Mechanism of Action
Binds the leptin receptor (LEPR) and activates JAK2-STAT3 signaling, regulating appetite, energy expenditure, and neuroendocrine function.
Routes of Administration
Goals & Uses
- Generalized lipodystrophyMetabolic DiseaseHigh
- Congenital leptin deficiencyGenetic Metabolic DisorderHigh
- Treatment of generalized lipodystrophyMetabolic DiseaseHigh
- Neuroendocrine restoration in hypothalamic amenorrheaReproductive EndocrinologyModerate
- Appetite suppression / weight managementObesityLow
- Partial lipodystrophy managementMetabolic DiseaseModerate
- Congenital leptin deficiency treatmentRare Genetic DiseaseModerate
- Immune modulationImmunologyLow
Contraindications
- Active malignancyOncologyHighUse caution or avoid depending on agent and context
- General obesity without lipodystrophyOff Label Use RiskHigh
- Hypersensitivity to metreleptinAllergyHigh
- PregnancyPopulationModeratePotential fetal risk or insufficient safety data
- Anti‑leptin antibodiesImmunologicModerate
Adverse Effects
- HypoglycemiaMetabolicUncommonAbnormally low blood glucose
- Injection site reactionsLocalCommon
- HeadacheNeurologicCommonPain in the head or upper neck
- Anti-metreleptin antibodiesImmunogenicityCommon
- Weight loss / decreased appetiteMetabolicCommon
- Injection site reactionLocalCommonRedness, swelling, itching, bruising, or pain at the injection site
- LymphomaOncologyRare
- Anti‑leptin antibody formationImmunologicRare
Drug Interactions
- InsulinModerateMay increase risk of low blood sugar
- ThiazolidinedionesLow
- SulfonylureasModerateMay increase risk of low blood sugar
Population Constraints
- PregnancyReproductive SafetyRelative
- Hepatic impairmentOrgan FunctionRelative
- Renal impairmentOrgan ImpairmentRelative
- Obese patients without leptin deficiencyMetabolicRelative
- Patients with congenital leptin deficiencyGeneticAbsolute
- Pediatric patients (<2 years)AgeRelative
Regulatory Status
- European UnionApprovedApproved: Generalized lipodystrophy, Leptin deficiencyApproved under EMA.
- United StatesApprovedApproved: Congenital leptin deficiency, Generalized lipodystrophyMetreleptin (Myalept) FDA‑approved.
- United KingdomApprovedApproved: Generalized lipodystrophy, Leptin deficiencyMHRA approval.
Metreleptin (Myalept) is FDA‑approved for leptin deficiency and generalized lipodystrophy; other indications remain investigational.
Evidence & Sources
No sources recorded yet.
Frequently Asked Questions
What is Leptin?
Leptin is a 167‑amino‑acid peptide hormone secreted by adipocytes that signals nutritional status to the hypothalamus. Recombinant human leptin (metreleptin) is used clinically for congenital leptin deficiency and generalized lipodystrophy.
What is Leptin used for?
Leptin is educationally associated with: Generalized lipodystrophy, Congenital leptin deficiency, Treatment of generalized lipodystrophy, Neuroendocrine restoration in hypothalamic amenorrhea, Appetite suppression / weight management, Partial lipodystrophy management, Congenital leptin deficiency treatment, Immune modulation. Educational only — not medical advice.
How is Leptin administered?
Recorded routes of administration: Intravenous, Subcutaneous.
What are the potential side effects of Leptin?
Reported adverse effects include: Hypoglycemia, Injection site reactions, Headache, Anti-metreleptin antibodies, Weight loss / decreased appetite, Injection site reaction, Lymphoma, Anti‑leptin antibody formation. This list is not exhaustive — consult a qualified clinician.
Who should avoid Leptin?
Recorded contraindications: Active malignancy, General obesity without lipodystrophy, Hypersensitivity to metreleptin, Pregnancy, Anti‑leptin antibodies. Consult a qualified clinician before use.