Details of Disease

General Information of Disease (ID: DIS1EPQ3)

• Disease Name: Congenital hyperinsulinism
• Synonyms: hyperinsulinemic hypoglycemia familial; hyperinsulinism congenital; hypoglycemia hyperinsulinemic of infancy; hyperinsulinism familial with pancreatic nesidioblastosis; congenital hyperinsulinism; persistent hyperinsulinemic hypoglycemia of infancy; Congenital Hyperinsulinism; PHHI; chi
• Disease Class: 5A4Y: Pancreatic internal secretion disorder
• Definition: Congenital isolated hyperinsulinism (CHI), a rare endocrine disease is the most frequent cause of severe and persistent hypoglycemia in the neonatal period and early infancy and is characterized by an excessive or uncontrolled insulin secretion (inappropriate for the level of glycemia) and recurrent episodes of profound hypoglycemia requiring rapid and intensive treatment to prevent neurological sequelae. CHI comprises 2 different forms: diazoxide-sensitive diffuse hyperinsulinism and diazoxide-resistant hyperinsulinism.
• Disease Hierarchy:
  DISYT3QM: Islet cell adenomatosis
  DISHQKQE: Familial hyperinsulinism
  DISHK54G: Overgrowth syndrome
  DIS1EPQ3: Congenital hyperinsulinism
• ICD Code: ICD-11: ICD-11: 5A4Y

Drug-Interaction Atlas (DIA) of This Disease

This Disease is Treated as An Indication in 1 Approved Drug(s)

Drug Name	Drug ID	Highest Status	Drug Type	REF
Diazoxide	DML1538	Approved	Small molecular drug	[1]

This Disease is Treated as An Indication in 3 Clinical Trial Drug(s)

Drug Name	Drug ID	Highest Status	Drug Type	REF
Avexitide	DMQCXSG	Phase 2	Peptide	[2]
RZ358	DMN32UV	Phase 2	Antibody	[3]
XOMA 358	DM5HXBC	Phase 2	NA	[4]

Molecular Interaction Atlas (MIA) of This Disease

References

• [1] Diazoxide FDA Label
• [2] ClinicalTrials.gov (NCT04652479) A Phase 2, Open-Label, Cross-over Study to Assess the Safety and Efficacy of Avexitide in Acquired Hyperinsulinemic Hypoglycemia. U.S.National Institutes of Health.
• [3] ClinicalTrials.gov (NCT04538989) An Open-Label Multiple-Dose Study of RZ358 in Patients With Congenital Hyperinsulinism. U.S.National Institutes of Health.
• [4] Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)