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Drug

Metreleptin

Approved · EMA
Late-stage development
Also known as Myalept, N-Methionylleptin, Metreleptina, Métréleptine+1 more

Myalept, N-Methionylleptin, Metreleptina, Métréleptine, Metreleptinum.

RxNorm1491625UNIITL60C27RLH
9
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy

Clinical trial2026-07-21Primary completion · ClinicalTrials.gov

Compassionate Use of Metreleptin in Previously-Treated Patients With Partial Lipodystrophy

Clinical trial2026-07-10Results expected Q3 2027 · ClinicalTrials.gov

Compassionate Use of Metreleptin in Previously-Treated Patients With Generalized Lipodystrophy

Clinical trial2026-07-08Results expected Q3 2027 · ClinicalTrials.gov

An Open-label Extension of APG-20 Study to Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy

Clinical trial2026-02-02Results expected Q1 2028 · ClinicalTrials.gov

Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia

Clinical trial2026-01-20Results expected Q3 2026 · ClinicalTrials.gov

Metreleptin in Anorexia Nervosa, Randomized Controlled Trial; Effects on Depressive Symptoms and Concomitant Changes in Brain Connectivity

Clinical trial2026-01-15Results expected Q3 2026 · ClinicalTrials.gov

A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized Lipodystrophy

Clinical trial2025-11-26Results posted · ClinicalTrials.gov

A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy

Clinical trial2025-07-03Results expected Q1 2028 · ClinicalTrials.gov

Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)

Clinical trial2025-06-12Results posted · ClinicalTrials.gov

Phase II Trial of Effect of Metreleptin Therapy in Severe Insulin Resistance

Clinical trial2025-01-30Results posted · ClinicalTrials.gov

CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Recombinant Human Leptin (METRELEPTIN) in Various Forms of Partial Lipodystrophy

Clinical trial2020-11-24Results posted · ClinicalTrials.gov

Approval: Myalepta (EMA)

Regulatory2018-07-30EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Metreleptin
Aliases & brands
MyaleptN-MethionylleptinMetreleptinaMétréleptineMetreleptinum
RxNorm CUI
1491625
UNII
TL60C27RLH
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2018-07-30
Latest approval
2018-07-30
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2018-07-30Approval
Approval: Myalepta (EMA)
Indication: Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed congenital… Show full indication

Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

18 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
18
registered trials across all phases
LATEST COMPLETION 2024
9
Active studies
4
Recruiting
7
Late-stage (III+)
9
Completed
PHASE DISTRIBUTIONn = 18
Phase 211Phase 34Phase 42Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy

RecruitingPH IVAmryt Pharman=122028

A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized Lipodystrophy

CompletedPH IVAegerion Pharmaceuticals, Inc.n=112024

An Open-label Extension of APG-20 Study to Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy

RecruitingPH IIIAmryt Pharman=242028

Compassionate Use of Metreleptin in Previously-Treated Patients With Generalized Lipodystrophy

Active, not recruitingPH IIINational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)n=242027

A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy

Active, not recruitingPH IIIChiesi Farmaceutici S.p.A.n=692026

Recent completions

Trials that read out recently, adding to the completed evidence base.

A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized Lipodystrophy

CompletedPH IVAegerion Pharmaceuticals, Inc.n=112024

CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Recombinant Human Leptin (METRELEPTIN) in Various Forms of Partial Lipodystrophy

CompletedPH IIUniversity of Michigann=112019

Short Term Effects of Leptin Withdrawal or Initiation in Lipodystrophy Independent of Energy Intake

CompletedPH IINational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)n=252018

Clinical Protocol to Investigate the Efficacy of Recombinant Human Leptin (Metreleptin) in Nonalcoholic Steatohepatitis (NASH) or Nonalcoholic Fatty Liver Disease (NAFLD) Associated With Lipodystrophy

CompletedPH IIUniversity of Michigann=232016

Long-Term Efficacy of Leptin Replacement in Treatment of Lipodystrophy

CompletedPH IINational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)n=1032015

An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy

CompletedN/ABristol-Myers Squibbn=412015
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.