Hyperinsulinism
Recent clinical, regulatory, research and industry developments relating to this disease.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 3 clinical trials expected to report results, the earliest in Q4 2026.
- Q4 202618F-Fluoro-L- DOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism
- Q3 2027A Phase 3, Single-Arm, Open-label, Pivotal Study to Evaluate the Efficacy and Safety of Ersodetug Compared to Baseline in Patients With Inadequately Controlled Hypoglycemia Due to Tumor Hyperinsulinism (Tumor HI)
- Q1 2030Use of18F-DOPA PET/MRI for the Detection and Localization of Focal Forms of Hyperinsulinism
Clinical MilestonesViewHide
- 2026-08-17A Phase 3, Single-Arm, Open-label, Pivotal Study to Evaluate the Efficacy and Safety of Ersodetug Compared to Baseline in Patients With Inadequately Controlled Hypoglycemia Due to Tumor Hyperinsulinism (Tumor HI)Results expected Q3 2027
- 2026-05-0118F-Fluoro-L- DOPA PET Imaging for the Detection and Localization of Focal Congenital HyperinsulinismResults expected Q4 2026
- 2025-12-19Use of18F-DOPA PET/MRI for the Detection and Localization of Focal Forms of HyperinsulinismResults expected Q1 2030
- 2026-07-01A Phase 2, Multiple Ascending Dose, Open-label, Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)Primary completion
- 2025-11-14A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study to Evaluate the Efficacy and Safety of RZ358 in Patients With Congenital HyperinsulinismPrimary completion
- 2030-12-30ClinicalInhibiting GABA Transaminase to Relieve Obesity Induced Hyperinsulinemia and Insulin ResistanceWithdrawn
- 2026-08-17ClinicalA Phase 3, Single-Arm, Open-label, Pivotal Study to Evaluate the Efficacy and Safety of Ersodetug Compared to Baseline in Patients With Inadequately Controlled Hypoglycemia Due to Tumor Hyperinsulinism (Tumor HI)Results expected Q3 2027
- 2026-07-01ClinicalA Phase 2, Multiple Ascending Dose, Open-label, Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of HM15136 (Efpegerglucagon) Treatment for 8 Weeks in Subjects Aged ≥2 Years With Congenital Hyperinsulinism (CHI)Primary completion
- 2026-05-01Clinical18F-Fluoro-L- DOPA PET Imaging for the Detection and Localization of Focal Congenital HyperinsulinismResults expected Q4 2026
- 2025-12-19ClinicalUse of18F-DOPA PET/MRI for the Detection and Localization of Focal Forms of HyperinsulinismResults expected Q1 2030
- 2025-11-14ClinicalA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study to Evaluate the Efficacy and Safety of RZ358 in Patients With Congenital HyperinsulinismPrimary completion
Research-associated treatments
Drugs and agents co-studied with this disease across the research literature — associative, not necessarily established treatments. Number shows shared papers.
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes4
- Hyperandrogenism1
- Hyperinsulinism1
- Insulin Resistance1
- Polycystic Ovary Syndrome1
Leading journals4
- Circulation1
- Endocrine reviews1
- Journal of ovarian research1
- The Journal of clinical endocrinology and metabolism1
Leading researchers8
- Behle K1
- Cheng X1
- Doverspike A1
- Ehrmann DA1
- Elahi D1
- He B1
- Heimesaat MM1
- Hentosz T1
Affiliations (unnormalised)4
- Allegheny General Hospital1
- Medizinische Universitäts-Klinik1
- Shengjing Hospital1
- The University of Chicago Pritzker School of Medicine1
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Reference
Authoritative identity, definition & identifiers.
A syndrome with excessively high INSULIN levels in the BLOOD. It may cause HYPOGLYCEMIA. Etiology of hyperinsulinism varies, including hypersecretion of a beta cell tumor (INSULINOMA); autoantibodies against insulin (INSULIN ANTIBODIES); defective insulin receptor (INSULIN RESISTANCE); or overuse of exogenous insulin or HYPOGLYCEMIC AGENTS.
- 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.