Lipodystrophy
Recent clinical, regulatory, research and industry developments relating to this disease.
The Causal Role of Ectopic Fat Deposition in the Pathogenesis of Metabolic Syndrome.
Altered adipose tissue and adipocyte function in the pathogenesis of metabolic syndrome.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 4 clinical trials expected to report results, the earliest in Q3 2027.
- Q3 2027Compassionate Use of Metreleptin in Previously-Treated Patients With Generalized Lipodystrophy
- Q3 2027Compassionate Use of Metreleptin in Previously-Treated Patients With Partial Lipodystrophy
- Q1 2028An 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
- Q2 2028A Two-Part, Randomized, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Pharmacokinetics of Mibavademab in Patients With Generalized Lipodystrophy (LAGO)
Clinical MilestonesViewHide
- 2026-07-28Compassionate Use of Metreleptin in Previously-Treated Patients With Partial LipodystrophyResults expected Q3 2027
- 2026-07-17Compassionate Use of Metreleptin in Previously-Treated Patients With Generalized LipodystrophyResults expected Q3 2027
- 2026-07-16An Open-label Extension of APG-20 Study to Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial LipodystrophyResults expected Q1 2028
- 2026-07-13A Two-Part, Randomized, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Pharmacokinetics of Mibavademab in Patients With Generalized Lipodystrophy (LAGO)Results expected Q2 2028
- 2026-07-21A 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 LipodystrophyPrimary completion
- 2026-07-09A Single-Arm, Open-Label, Safety Study in Patients With Generalized Lipodystrophy Switching From Metreleptin to Mibavademab, A Leptin Receptor Agonist AntibodyCompleted
- 2026-07-28ClinicalCompassionate Use of Metreleptin in Previously-Treated Patients With Partial LipodystrophyResults expected Q3 2027
- 2026-07-21ClinicalA 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 LipodystrophyPrimary completion
- 2026-07-17ClinicalCompassionate Use of Metreleptin in Previously-Treated Patients With Generalized LipodystrophyResults expected Q3 2027
- 2026-07-16ClinicalAn Open-label Extension of APG-20 Study to Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial LipodystrophyResults expected Q1 2028
- 2026-07-13ClinicalA Two-Part, Randomized, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Pharmacokinetics of Mibavademab in Patients With Generalized Lipodystrophy (LAGO)Results expected Q2 2028
- 2026-07-09ClinicalA Single-Arm, Open-Label, Safety Study in Patients With Generalized Lipodystrophy Switching From Metreleptin to Mibavademab, A Leptin Receptor Agonist AntibodyCompleted
- 2025-11-26ClinicalA 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized LipodystrophyResults posted
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approval — Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complic… (2018)
Clinical trials
The current development programme across all trial phases.
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes2
- Insulin Resistance1
- Metabolic Syndrome1
Leading journals2
- International journal of molecular sciences1
- The Journal of clinical investigation1
Leading researchers4
- Janssen JAMJL1
- Kahn CR1
- Lee KY1
- Wang G1
Affiliations (unnormalised)4
- Erasmus Medical Center (Erasmus MC)1
- Heritage College of Osteopathic Medicine1
- Joslin Diabetes Center1
- The Diabetes Institute1
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Reference
Authoritative identity, definition & identifiers.
A collection of heterogenous conditions resulting from defective LIPID METABOLISM and characterized by ADIPOSE TISSUE atrophy. Often there is redistribution of body fat resulting in peripheral fat wasting and central adiposity. They include generalized, localized, congenital, and acquired lipodystrophy.
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.