FDA nudges development of drug to prevent necrotizing enterocolitis

Key takeaways:

• Infinant Health is investigating a live biotherapeutic product to prevent necrotizing enterocolitis in preterm babies.
• Trials of a similar product showed a 73% lower risk for the condition among very low-birth-weight infants.

The FDA recently granted orphan drug and rare pediatric disease designations to Infinant Health for a live biotherapeutic product to prevent necrotizing enterocolitis in preterm infants, the company announced.

The drug candidate, INF108, includes a strain of Bifidobacterium longum subspecies infantis.

Arlene Fosmer, MS

“It works together with breast milk to colonize and protect the gastrointestinal tract of preterm infants, reducing the levels of inflammation and potentially pathogenic bacteria in the infant’s gut,” Arlene Fosmer, MS, Infinant Health’s chief scientific officer, told Healio.

The company applied for orphan drug designation based on studies of a similar product, EVC001, which is sold under the brand name Evivo. In a cohort of 483 infants, EVC001 appeared to reduce the risk for developing necrotizing enterocolitis (NEC) by 73% among very-low-birth-weight infants (adjusted risk ratio = 0.27; 95% CI, 0.094-0.614).

Neonatologists previously used Bifidobacterium infantis probiotic products off-label to prevent preterm babies from developing NEC, but the FDA instructed physicians to stop doing so in 2023 after an infant died who was subsequently found to have Evivo B. infantis in their blood culture.

Brian Scottoline, MD, PhD

Brian Scottoline, MD, PhD, professor in the division of neonatology at the Oregon Health and Science University School of Medicine and a medical adviser assisting with Infinant Health’s upcoming trials, told Healio that the FDA’s instructions “caused a fair amount of concern” among neonatologists.

Additionally, he said the risk for bacteremia or sepsis appears to be very low. A recent systematic review and meta-analysis showed that probiotic sepsis occurred in just eight out of 20,000 infants who received probiotics in the NICU, which equates to one in every 2,500 infants. The authors pointed out that for every baby who developed probiotic sepsis, 92 infants who did not receive probiotics were also diagnosed with sepsis, “suggesting consideration of risk-benefit may be warranted.”

“There are a substantial number of neonatologists who remain very interested in the potential for B. infantis to prevent NEC in their NICUs, pending successful trial results, safety data and approval by the FDA,” Scottoline said.

Scottoline said the company is taking several measures to ensure safety during upcoming clinical trials for INF108, including testing every batch to verify that there are no other organisms, and having clear sepsis detection guidelines and protocols in the event an infant does develop a related infection.

Fosmer explained that although INF108 is similar to EVC001, it will be manufactured in an FDA-compliant pharmaceutical facility with processes to ensure the product is as consistent and pure as possible. Because of this, she said the FDA considers them two distinct products. She also noted that EVC001 is a dietary supplement for full-term infants and is not intended to treat NEC.

Orphan drug designation waives the FDA’s biologics license application fee, which Fosmer said will save the company more than $4 million, and pending successful clinical trials, the designation also offers the opportunity for market exclusivity.

In the past, the FDA could issue a priority review voucher to drug applications with rare pediatric disease designation, but the program expired in December 2024.

“Orphan drug designation and rare pediatric disease designation enhance investor confidence in our company, facilitating the fundraising necessary to advance our pharmaceutical product development, including conducting a phase 1 safety trial, followed by a phase 2/3 efficacy trial,” Fosmer said.

Fosmer said the company is planning to launch a phase 1 safety trial in early 2026.

“We appreciate the unwavering support from advocacy groups like the NEC Society, patient families and providers across the United States who have been instrumental in supporting this journey and advancing our mission,” Fosmer said.

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For more information:

Arlene Fosmer, MS, can be reached at afosmer@infinanthealth.com.
Brian Scottoline, MD, PhD, can be reached at scottoli@ohsu.edu.