INF108 Granted Orphan Drug Designation, Rare Pediatric Drug Designation for Preterm Infants With NEC

The FDA granted an orphan drug designation (ODD) and a rare pediatric disease designation (RPDD) for INF108 (Infinant Health) for the prevention of necrotizing enterocolitis (NEC) in preterm infants. The manufacturer is planning to initiate a human clinical trial with the patented investigational drug, which is a strain of Bifidobacterium longum subspecies infantis.¹

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NEC is a life-threatening illness with a mortality rate as high as 50% that almost exclusively affects neonates, according to the National Institutes of Health. The pathophysiology of NEC is inflammation of the intestinal lining, which leads to bacterial invasion, causing cellular damage, cellular death, and necrosis of the colon and intestine. As the disease progresses, it can lead to intestinal perforation, causing peritonitis, sepsis, and death. Symptoms are nonspecific and may include vomiting, lethargy, abdominal tenderness, and poor feeding.²

Currently, INF108 is not commercially available. Bifidobacterium infantis (B. infantis) is well-adapted to work with human milk to establish the infant gut microbiome, allowing it to break down human milk oligosaccharides (HMOs), which are otherwise undigestible by the infant. Prior clinical evidence suggests that B. infantis is associated with a significant reduction in the incidence of NEC and NEC-related mortality in very low birth weight infants. The microbiomes of preterm infants given B. infantis also contain lower levels of antibiotic-resistant organisms and show biomarkers for reduced gut inflammation.^1,3

Further, INF108 stems from the discovery of the bacterial strain of B. infantis, EVC001, which has clinically demonstrated to have all genes required to metabolize HMOs within breast milk. The strain is specifically formulated to prevent NEC. By utilizing HMOs, INF108 can replicate and persist—or colonize—within the infant gut. Unlike other probiotic strains, INF108 does not pass through and instead maintains a presence in the gut. There has been no other clinical study that has demonstrated both a substantial and persistent beneficial change in the gut microbiome, according to the manufacturer’s website.⁴

Additionally, INF108 can convert HMOs into key signaling molecules called metabolites that help guide healthy immune system function. This then helps promote a healthy functioning gut and “teaches” immune cells to not become cells that would overreact when infants are exposed to new foods.⁴

An ODD is granted to therapies that are intended to treat, diagnose, or prevent rare diseases or disorders that affect less than 200,000 people in the US. Additionally, an RPD grants the manufacturer a priority review voucher, with the goal that INF108 is approved before the program “sunsets” in September 2026.¹

"Securing this ODD sends a positive message to the neonatology community," Mike Johnson, CEO of Infinant Health, said in a news release. "The RPDD 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

REFERENCES

1. PR Newswire. FDA Grants Orphan Drug & Rare Pediatric Disease Designations to Infinant Health for NEC. News release. May 7, 2025. Accessed May 7, 2025. https://www.prnewswire.com/news-releases/fda-grants-orphan-drug--rare-pediatric-disease-designations-to-infinant-health-for-nec-302447903.html

2. National Library of Medicine — National Center for Biotechnology Information. Necrotizing Enterocolitis — Continuing Education Activity. Updated August 8, 2023. Accessed May 7, 2025. https://www.ncbi.nlm.nih.gov/books/NBK513357/#:~:text=Introduction-,Necrotizing%20enterocolitis%20(NEC)%20is%20a%20life%2Dthreatening%20illness%20almost,of%20the%20colon%20and%20intestine

3. BioSpace. Infinant Health Announces Submission of Orphan Drug Designation Application. News release. April 2, 2024. Accessed May 7, 2025. https://www.biospace.com/infinant-health-announces-submission-of-orphan-drug-designation-application

4. Infinant Health. Leveraging the microbiome for better health. Accessed May 7, 2025. https://www.infinanthealth.com/about/our-science