Disease

Transient neonatal diabetes mellitus

Late-stage therapeutic development

Also known as TNDM, transient neonatal diabetes mellitus (disease), chromosome 6-associated transient diabetes mellitus, diabetes mellitus, 6q24-related transient neonatal+1 more

TNDM, transient neonatal diabetes mellitus (disease), chromosome 6-associated transient diabetes mellitus, diabetes mellitus, 6q24-related transient neonatal, diabetes mellitus, transient neonatal.

Clinical trials

Associated genes

Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate

Key developments

6 clinical trials expected to report results, the earliest in Q1 2027.

Upcoming Milestones

Major developments

Upcoming trial readoutHigh impact

A Randomized, Double-Masked, Multi-Center, 3-Arm Pivotal Phase 2/3 Study to Evaluate The Efficacy and Safety of Intravitreal EYE103 Compared With Intravitreal Ranibizumab (0.5mg) in Participants With Diabetic Macular Edema

Results expected Q1 20272026-06-26

Upcoming trial readoutHigh impact

Efficacy and Safety of Cagrilintide for Weight Management in Participants With Overweight or Obesity and Type 2 Diabetes

Results expected Q2 20272026-04-21

Clinical Milestones13View all 13

Results posted 1

2026-01-06Efficacy and Safety of Teneligliptin in Combination With Metformin in Chinese Patients With Type 2 Diabetes MellitusResults posted

Results expected 5

2026-06-26A Randomized, Double-Masked, Multi-Center, 3-Arm Pivotal Phase 2/3 Study to Evaluate The Efficacy and Safety of Intravitreal EYE103 Compared With Intravitreal Ranibizumab (0.5mg) in Participants With Diabetic Macular EdemaResults expected Q1 2027
2026-04-21Efficacy and Safety of Cagrilintide for Weight Management in Participants With Overweight or Obesity and Type 2 DiabetesResults expected Q2 2027
2026-04-20A Master Protocol to Investigate the Efficacy and Safety of Orforglipron Tablet Once Daily Compared With Placebo in Participants With Obesity or Overweight With and Without Type 2 DiabetesResults expected Q3 2027
2026-03-18A 96-week, Single-arm Study to Evaluate the Efficacy and Safety of 8mg Aflibercept in Subjects With Proliferative Diabetic Retinopathy (PDR) Without Center-involved Diabetic Macular Edema (DME)Results expected Q1 2028
2026-01-13Semaglutide for Dialysis-Treated Patients - a Glucose Time in Range Study- DIALYSIS-TIR StudyResults expected Q3 2027

Other updates 5

+5 more in the activity timeline below

Activity timeline13

2029-02-28ClinicalEvaluate the Efficacy and Safety of the Combined Use of a Sodium-glucose Cotransporter 2 (iSGLT2) Inhibitor and a Dipeptidyl Peptidase-4 (iDPP -4) Inhibitor in the Treatment of Type 2 Diabetes Mellitus in Patients Treated With MetforminWithdrawn
2028-07-31ClinicalIntervention for High-normal Blood Pressure in Adults With Type 2 Diabetes-----renal SubstudyWithdrawn
2028-03-01ClinicalA Phase 3, Prospective, Adaptive, Randomized, Double-blind, Triple-masked, Parallel, Multicenter Clinical Trial to Evaluate the Efficacy and Safety of the N0750 Compared to Monotherapies in the Treatment of Painful Diabetic Peripheral NeuropathyWithdrawn
2028-02-01ClinicalPhase III, Adaptive, Multicenter, Randomized, Superiority, Double-Dummy, Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of a Fixed-Dose Combination of a Sodium-Glucose Cotransporter-2 (SGLT2) Inhibitor and a Thiazolidinedione in the Treatment of Type 2 Diabetes Mellitus, Compared to Monotherapy, in Patients Treated With MetforminWithdrawn
2027-01-01ClinicalComparison of Subvisible Retinal Laser Therapy With Intravitreal Bevacizumab in Treatment of Diabetic Macular EdemaWithdrawn
2026-06-30ClinicalMetformin Versus Standard of Care Treatment in Pregnant Women With Prediabetes: A Randomized TrialWithdrawn
2026-06-26ClinicalA Randomized, Double-Masked, Multi-Center, 3-Arm Pivotal Phase 2/3 Study to Evaluate The Efficacy and Safety of Intravitreal EYE103 Compared With Intravitreal Ranibizumab (0.5mg) in Participants With Diabetic Macular EdemaResults expected Q1 2027
2026-04-21ClinicalEfficacy and Safety of Cagrilintide for Weight Management in Participants With Overweight or Obesity and Type 2 DiabetesResults expected Q2 2027
2026-04-20ClinicalA Master Protocol to Investigate the Efficacy and Safety of Orforglipron Tablet Once Daily Compared With Placebo in Participants With Obesity or Overweight With and Without Type 2 DiabetesResults expected Q3 2027
2026-03-18ClinicalA 96-week, Single-arm Study to Evaluate the Efficacy and Safety of 8mg Aflibercept in Subjects With Proliferative Diabetic Retinopathy (PDR) Without Center-involved Diabetic Macular Edema (DME)Results expected Q1 2028

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Glyburideapproved

Approval — Amglidia is indicated for the treatment of neonatal diabetes mellitus, for use in newborn… (2018)

Clinical trials

15 sponsors · 3 new · 0 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

Semaglutide for Dialysis-Treated Patients - a Glucose Time in Range Study- DIALYSIS-TIR Study

Phase 4 · Active, not recruiting · University of Texas Southwestern Medical Center

A 96-week, Single-arm Study to Evaluate the Efficacy and Safety of 8mg Aflibercept in Subjects With Proliferative Diabetic Retinopathy (PDR) Without Center-involved Diabetic Macular Edema (DME)

Phase 4 · Active, not recruiting · Edward Wood, MD

Phase 2/3 · Active, not recruiting · EyeBiotech Ltd.

Beta Cell Function in Type 2 Diabetes in Black Patients: Differential Effects of SGLT2 Inhibitors and GLIP-Receptor Agonists

Phase 4 · Active, not recruiting · MaryAnn Banerji

A Master Protocol to Investigate the Efficacy and Safety of Orforglipron Tablet Once Daily Compared With Placebo in Participants With Obesity or Overweight With and Without Type 2 Diabetes

Phase 3 · Active, not recruiting · Eli Lilly and Company

Efficacy and Safety of Cagrilintide for Weight Management in Participants With Overweight or Obesity and Type 2 Diabetes

Phase 3 · Active, not recruiting · Novo Nordisk A/S

Recently completed

Efficacy and Safety of Teneligliptin in Combination With Metformin in Chinese Patients With Type 2 Diabetes Mellitus

Phase 3 · Completed · Tanabe Pharma Corporation

Fluocinolone Acetonide in Diabetic Macular Edema (FAME) Extension Study

Phase 3 · Completed · Alimera Sciences

Effect of Rubus Occidentalis Extract on Plasma Glucose Levels in Subject With Prediabetes; A Proof-of-concept, Randomized, Double-blind, Parallel-group Placebo-controlled Study

Phase 4 · Completed · Korea University Anam Hospital

A Phase III, Randomised, Double-blind, Placebo-controlled Parallel Group Safety and Efficacy Study of Linagliptin (5 mg Administered Orally Once Daily) Over 12 Weeks Followed by a 40 Week Double-blind Extension Period (Placebo Patients Switched to Glimepiride) in Drug Naive or Previously Treated Type 2 Diabetic Patients With Moderate to Severe Renal Impairment and Insufficient Glycaemic Control

Phase 3 · Completed · Boehringer Ingelheim

A Randomised, Double-blind, Placebo-controlled Parallel Group Efficacy and Safety Trial of BI 10773 (10 and 25 mg Administered Orally Once Daily) Over 24 Weeks in Patients With Type 2 Diabetes Mellitus With Insufficient Glycaemic Control Despite a Background Therapy of Pioglitazone Alone or in Combination With Metformin

Phase 3 · Completed · Boehringer Ingelheim

An Open-label, Randomized and Crossover Study to Assess the Effect of Co-administration of Vildagliptin and Voglibose on the Steady-state Pharmacokinetics / Pharmacodynamics in Japanese Patients With Type 2 Diabetes

Phase 4 · Completed · Novartis Pharmaceuticals

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals

2018emaApproval — Glyburide· Amglidia is indicated for the treatment of neonatal diabetes mellitus, for use in newborns, infants and children. Sulphonylureas like Amglidia have been shown to be effective in patients with mutations in the genes coding for the ?-cell ATP-sensitive potassium channel and chromosome 6q24-related transient neonatal diabetes mellitus. source ↗

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.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

ZFP570.81 KCNJ110.72 ABCC80.70 MOG0.54 PLAGL10.48 SLC2A20.39 LIPE0.08 GPN10.07 GIMAP60.07 AFMID0.07 DNAJC300.07 FAM186B0.07

Disease biology

3 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

ATP-sensitive inward rectifier potassium channel 11via KCNJ110.72 ATP-binding cassette sub-family C member 8via ABCC80.70 Myelin-oligodendrocyte glycoproteinvia MOG0.54

Reference

Authoritative identity, definition & identifiers.

Synonyms

TNDM, transient neonatal diabetes mellitus (disease), chromosome 6-associated transient diabetes mellitus, diabetes mellitus, 6q24-related transient neonatal, diabetes mellitus, transient neonatal

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Related entities are derived from literature co-mention (studied together) — associative, not causal.