

Drug discovery
14
drugs
With orphan designations
Overview
Necrotizing enterocolitis (NEC) is a life-threatening gastrointestinal emergency most common in preterm infants, characterized by intestinal inflammation, mucosal injury, and necrosis. Pathogenesis involves intestinal ischemia, dysbiosis, and enteral feeding in immature gut mucosa. Mortality approaches 20–30%, with survivors at risk for short bowel syndrome, neurodevelopmental delays, and intestinal strictures [1][5][6].
Burden
Mortality rates reach 50% in surgical cases; survivors face 30–50% risk of long-term neurodevelopmental/GI sequelae [1][6].
Annual U.S. hospitalization costs exceed $1 billion, with incremental post-discharge costs up to $18,274 per infant [13].
Leading cause of infant mortality among GI disorders, contributing to 10.2 deaths per 100,000 live births [12][13].
Therapies
Medical: NPO status, gastric decompression, IV antibiotics (ampicillin + aminoglycoside ± anaerobic coverage), and parenteral nutrition [1][6].
Surgical: Laparotomy with bowel resection for perforation or necrosis (required in 25–40% of cases) [1][6].
Emerging: Probiotics, stem cell therapy (BM-MSCs/AF-MSCs), and IL-22 supplementation show experimental promise [5][15].
Categories: rare gastroenterological diseases, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
bifidobacterium longum subsp. infantis | other | FDA | 2025-05-06 | — | Infinant Health, Inc. |
Amnion-derived Cellular Cytokine Solution | cell therapies | FDA | 2022-04-26 | — | Noveome Biotherapeutics, Inc. |
Human Placental Extract | other | FDA | 2020-05-28 | — | Plakous Therapeutics, Inc. |
inter-Alpha-Inhibitor-Proteins (IaIp) (Human) | proteins | FDA | 2018-02-13 | — | ProThera Biologics, Inc. |
melatonin | small molecules | FDA | 2017-01-25 | — | WORPHMED Srl |
Melatonin | small molecules | EMA | 2016-08-01 | — | Worphmed Srl |
Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis | combination | FDA | 2015-03-24 | — | Leadiant Biosciences, Inc. |
bovine lactoferrin | proteins | FDA | 2015-02-23 | — | Metrodora Therapeutics, LLC |
Lactobacillus reuteri | other | EMA | 2015-02-12 | — | Infant Bacterial Therapeutics AB |
Lactobacillus acidophilus and Bifidobacterium bifidum | other | EMA | 2013-12-18 | — | Laboratorio Farmaceutico S.I.T. s.r.l. |
L. reuteri | other | FDA | 2013-08-01 | — | Infant Bacterial Therapeutics |
Heparin-binding epidermal growth factor-like growth factor (HB-EGF), amino acids 74-148 | proteins | EMA | 2006-10-31 | — | Dr Michael Moore |
Heparin-binding epidermal growth factor-like growth factor | proteins | FDA | 2006-09-18 | — | Trillium Therapeutics, Inc. |
Bacitracin | — | FDA | 1984-03-13 | — | A. L. Laboratories, Inc. |