Metreleptin

Summary

Metreleptin (CHEMBL2107857) is an approved protein (ATC A16AA07); indicated across 13 conditions including lipodystrophy and liver disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Protein
• ATC class: A16AA07
• Indications: 13 conditions
• Clinical trials: 27

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2107857
Name	Metreleptin
Type	Protein
Max phase	4
ATC	A16AA07

Also known as: Leptin (human), n-methionyl-, Metreleptin, Metreleptina, Metreleptine, Myalept, Myalepta, N-methionylleptin (human), R-METHULEPTIN, METRELEPTIN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

13 indications (3 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
lipodystrophy	4	MONDO:0006573	EFO:1000727
liver disorder	4	MONDO:0005154	EFO:0001421
familial partial lipodystrophy	4	MONDO:0020088	MONDO:0020088
obesity disorder	3	MONDO:0011122	EFO:0001073
metabolic dysfunction-associated steatotic liver disease	2	MONDO:0013209	EFO:0003095
metabolic syndrome X	2	MONDO:0011565	EFO:0000195
lipomatosis	2	MONDO:0006574	EFO:1000728
amenorrhea	2	MONDO:0001836	MONDO:0001836
anorexia nervosa	2	MONDO:0005351	MONDO:0005351
type 1 diabetes mellitus	1	MONDO:0005147	MONDO:0005147

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 27.

Phase distribution

Phase	Trials
PHASE2	15
PHASE3	4
Not specified	4
PHASE4	2
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06484868	PHASE4	RECRUITING	Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
NCT04026178	PHASE4	COMPLETED	Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy
NCT02262832	PHASE3	ACTIVE_NOT_RECRUITING	Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy
NCT05164341	PHASE3	ACTIVE_NOT_RECRUITING	Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL
NCT06502990	PHASE3	RECRUITING	Open-label Study to Evaluate Metreleptin in Children Under 6 Years of Age With Generalised Lipodystrophy
NCT06679270	PHASE3	RECRUITING	Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy
NCT00085982	PHASE2	ACTIVE_NOT_RECRUITING	Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance
NCT02262806	PHASE2	ACTIVE_NOT_RECRUITING	Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy
NCT05351164	PHASE2	ACTIVE_NOT_RECRUITING	Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
NCT06305182	PHASE2	RECRUITING	Metreleptin in Anorexia Nervosa
NCT00025883	PHASE2	COMPLETED	Leptin to Treat Lipodystrophy
NCT00130117	PHASE2	COMPLETED	Study of Leptin for the Treatment of Hypothalamic Amenorrhea
NCT00140231	PHASE1/PHASE2	COMPLETED	Role of Leptin in the Neuroendocrine and Immune Response to Fasting
NCT00140244	PHASE2	COMPLETED	Randomized, Placebo-Controlled Study of Leptin for the Treatment of HIV Lipodystrophy and Metabolic Syndrome
NCT00392925	PHASE2	COMPLETED	A Study to Evaluate the Effect on Body Weight of Leptin Administered in Conjunction With Pramlintide in Overweight and Obese Subjects
NCT00596934	PHASE2	COMPLETED	Recombinant Leptin Therapy for Treatment of Nonalcoholic Steatohepatitis (NASH)
NCT00673387	PHASE2	COMPLETED	Study to Examine Safety, Tolerability, and Effect on Body Weight of Metreleptin Administered in Conjunction With Pramlintide in Obese and Overweight Subjects
NCT00819234	PHASE2	COMPLETED	Extension Study of Protocol DFA102 to Examine the Long-Term Safety, Tolerability, and Effect on Body Weight of Pramlintide Administered in Combination With Metreleptin
NCT01235741	PHASE2	TERMINATED	A Study To Examine The Efficacy And Safety Of Pramlintide+Metreleptin In Obese Subjects
NCT01679197	PHASE2	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
NCT01778556	PHASE2	COMPLETED	Short-term Effects of Leptin in People With Lipodystrophy
NCT02654977	PHASE2	COMPLETED	CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy
NCT00140205	PHASE1	COMPLETED	Pharmacokinetics of Leptin Administration During Fasting
NCT02325674	Not specified	RECRUITING	MEASuRE: Metreleptin Effectiveness And Safety Registry
NCT02404896	Not specified	AVAILABLE	Expanded Access Metreleptin Study
NCT00677313	Not specified	COMPLETED	An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy
NCT04063488	Not specified	COMPLETED	The Effects of Metreleptin in Congenital Leptin Deficiency

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).