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Myalept

(Metreleptin)

ApprovedSmall molecule

Chiesi Farmaceutici S.p.A.

Metreleptin is used to treat a rare condition where the body doesn't produce enough leptin, a hormone that helps regulate hunger and weight. It helps patients with this condition manage their weight and metabolism.[2] Myalept is currently approved and in use across 8 indications[4].[1]

Myalept is one of 129 small molecule programs targeting Lipodystrophy. It is currently in Approved.

8

Indications

21

Total Trials

9

Active Trials

128

Competitors

1

FDA Application

NDA/BLA/ANDA regulatory filings

Pipeline

Status by Indication[4][1]

Indication

LipodystrophyActive, not recruiting

DiabetesActive, not recruiting

Liver DiseaseUnknown

Familial Partial LipodystrophyRecruiting

HypertriglyceridemiaActive, not recruiting

Insulin ResistanceActive, not recruiting

Partial LipodystrophyRecruiting

LipomatosisActive, not recruiting

LipodystrophyPhase 3

Active, not recruiting

DiabetesPhase 2

Active, not recruiting

Liver DiseasePreclinical

Unknown

Familial Partial LipodystrophyPhase 3

Recruiting

HypertriglyceridemiaPreclinical

Active, not recruiting

Insulin ResistancePhase 2

Active, not recruiting

Partial LipodystrophyApproved

Recruiting

LipomatosisPhase 2

Active, not recruiting

Competition

Competitive Landscape

100 competitors in Liver Disease

#DrugCompanyPhaseTrials

Myalept

Chiesi Farmaceutici S.p.A.

Rydapt

Exjade

Fabhalta

Veozah

Tukysa

Incivek

Vertex Pharmaceuticals

Vanflyta

Tabrecta

Brintellix

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 3Active, not recruiting

Safety and Efficacy

NCT05164341

Phase 2Recruiting

Anorexia Nervosa

NCT06305182

Q3 2027

Phase 2Active, not recruiting

Diabetes

NCT02262806

Phase 3Active, not recruiting

Lipodystrophy

NCT02262832

Q1 2028

Post-ApprovalRecruiting

Partial Lipodystrophy

NCT06484868

Q4 2028

Phase 2Active, not recruiting

Lipomatosis

NCT05351164

Clinical

Trials by Indication[1]

Lipodystrophy3 trials

Short-term Effects of Leptin in People With Lipodystrophy

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT01778556·25 enrolled

Leptin to Treat Lipodystrophy

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT00025883·103 enrolled

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

Active, not recruiting·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 3·NCT02262832·24 enrolled

Partial Lipodystrophy2 trials

Open-label Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

Recruiting·AAmryt Pharma·Phase 4·NCT06484868·12 enrolled

CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy

Completed·UUniversity of Michigan·Phase 2·NCT02654977·11 enrolled

Overweight2 trials

Study to Examine Safety, Tolerability, and Effect on Body Weight of Metreleptin Administered in Conjunction With Pramlintide in Obese and Overweight Subjects

Completed· AstraZeneca logo

AstraZeneca·Phase 2·NCT00673387·636 enrolled

A Study to Evaluate the Effect on Body Weight of Leptin Administered in Conjunction With Pramlintide in Overweight and Obese Subjects

Completed· AstraZeneca logo

AstraZeneca·Phase 2·NCT00392925·177 enrolled

Generalized Lipodystrophy2 trials

Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy

Completed·AAegerion Pharmaceuticals, Inc.·Phase 4·NCT04026178·11 enrolled

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

Terminated·AAmryt Pharma·Phase 3·NCT06502990·1 enrolled

Hepatitis1 trial

Recombinant Leptin Therapy for Treatment of Nonalcoholic Steatohepatitis (NASH)

Completed·EElif Oral·Phase 2·NCT00596934·9 enrolled

10 more indications

Safety and Efficacy1 trial

Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL

Active, not recruiting·AAmryt Pharma·Phase 3·NCT05164341·69 enrolled

ESS1 trial

Expanded Access Metreleptin Study

Available·UUniversity of Michigan··NCT02404896

Insulin resistance1 trial

Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance

Active, not recruiting·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT00085982·11 enrolled

Alcoholic Fatty Liver Disease1 trial

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

Completed·UUniversity of Michigan·Phase 2·NCT01679197·23 enrolled

Lipomatosis1 trial

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

Active, not recruiting·UUniversity of Michigan·Phase 2·NCT05351164·4 enrolled

Diabetes1 trial

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

Active, not recruiting·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT02262806·29 enrolled

Anorexia Nervosa1 trial

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

Recruiting·GGabriella Milos·Phase 2·NCT06305182·50 enrolled

Familial Partial Lipodystrophy1 trial

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

Recruiting·AAmryt Pharma·Phase 3·NCT06679270·24 enrolled

Generalised Lipodystrophy1 trial

MEASuRE: Metreleptin Effectiveness And Safety Registry

Recruiting·AAmryt Pharma··NCT02325674·100 enrolled

Unspecified2 trials

The Effects of Metreleptin in Congenital Leptin Deficiency

Completed·NNorthwestern University··NCT04063488·2 enrolled

Expanded Access for Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy

No longer available· Bristol-Myers Squibb logo

Bristol-Myers Squibb··NCT00677313

Regulatory

FDA Approval Timeline[5]

Feb 24, 2014

MYALEPT

BLA BLA125390

Sources

References

[1]
ClinicalTrials.gov — 21 trial records for Metreleptin. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Myalept drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Metreleptin compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Metreleptin. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (BLA BLA125390). U.S. Food & Drug Administration.
[6]
SEC EDGAR — Chiesi Farmaceutici S.p.A. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Leptin Receptor Agonist[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: Chiesi Farmaceutici S.p.A.

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