INF108 to prevent necrotizing enterocolitis receives FDA designations | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

In a May 7, 2025 announcement, Infinant Health stated that its investigational drug candidate INF108 received both orphan drug and rare pediatric disease designations for the prevention of necrotizing enterocolitis (NEC) in preterm infants.¹

According to a press release from the company, NEC is a life-threatening condition marked by the death of intestinal tissue, which impacts preterm infants. NEC occurs when inflammation of the intestinal lining occurs and begins to deteriorate. The exact cause of the disease is unknown, however, it is believed that a decrease in blood flow to the bowel keeps it from producing mucus, which protects the gastrointestinal tract. Additionally, certain types of bacteria in the intestine can also result in NEC.

Prevalence of NEC

Infinant Health stated that NEC is a leading cause of death in preterm infants, impacting thousands each year. According to a 2020 study published in BMC Pediatrics, NEC is also a major cause of morbidity and mortality in the neonatal intensive care unit (NICU).^1,2

The study, led by Amer Alsaied, was a systematic review and meta-analysis of cohort studies reporting the incidence of NEC among infants with very low birth weight (VLBW). Following a comprehensive and systematic search of databases, the investigators identified 27 cohort studies that reported NEC incidence. Across these studies, there were 574,692 neonates, of whom 39,965 developed NEC. Incidence rates were pooled using random effect models (REM), in the presence of substantial heterogeneity. Pooled estimate of NEC, based on REM, was 7% (95% CI: 6.0–8.0%), according to BMC Pediatrics publication. In all, 7 out of 100 of all VLBW infants in the NICU were likely to develop NEC, though the study authors noted "considerable heterogeneity between studies."²

Impact of FDA designations

"Securing this Orphan Drug Designation sends a positive message to the neonatology community," said Mike Johnson, CEO of Infinant Health, in the announcement. "The Rare Pediatric Disease Designation is an important step toward securing a priority review voucher upon the approval of INF108, which will help accelerate our clinical development and bring hope to families facing limited options for NEC prevention."¹

Orphan drug designation is granted by the FDA to therapies intended to treat, prevent, or diagnose rare diseases or disorders that impact fewer than 200,000 people in the United States. Rare pediatric disease designation grants the company or manufacturer a priority review voucher if approval for INF108 is granted before the rare pediatric disease program ends. Should Congress not renew the program, it will "sunset" in September of 2026, according to Infinant Health.

The company plans to initiate a human clinical trial with INF108 for use in preterm infants.

References:

1. FDA grants orphan drug & rare pediatric disease designations to Infinant Health for NEC. Infinant Health. Press release. May 7, 2025. Accessed May 8, 2025. https://www.prnewswire.com/news-releases/fda-grants-orphan-drug--rare-pediatric-disease-designations-to-infinant-health-for-nec-302447903.html

2. Alsaied A, Islam N, Thalib L. Global incidence of Necrotizing Enterocolitis: a systematic review and Meta-analysis. BMC Pediatr. 2020 Jul 13;20(1):344.
doi:10.1186/s12887-020-02231-5. PMID: 32660457; PMCID: PMC7359006