Neurenati Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to NEU-001, a novel combination therapy for Hirschsprung disease. The dual designations mark a significant milestone in the development of what could become the first non-surgical treatment option for newborns with this life-threatening congenital gastrointestinal disorder.
NEU-001 utilizes a neurotrophic growth factor administered intrarectally to regenerate the enteric nervous system in newborns with Hirschsprung disease. The therapy aims to restore gastrointestinal motility after birth, potentially eliminating the need for invasive pull-through surgery, which is currently the standard of care.
"Currently, surgery remains the only option for newborns with Hirschsprung disease. Post-surgery, more than 40% of patients will endure various inflammatory bowel diseases, including enterocolitis and other complications, during all their lives," explained Dr. Maxime Ranger, PhD, MBA, CEO of Neurenati Therapeutics. "Our goal is to offer a non-surgical therapy aimed at promoting nerve cell formation to complete the development of the enteric nervous system."
Understanding Hirschsprung Disease
Hirschsprung disease is a life-threatening congenital gastrointestinal disorder caused by the absence of nerve cells in parts of the lower GI tract. According to Neurenati Therapeutics, approximately 80% of cases are diagnosed within the first four weeks of life.
The disease is classified into two types based on the length of the affected intestinal segment:
- Short segment (most common): Affects only the rectosigmoid colon
- Long segment: Extends beyond the sigmoid colon, sometimes reaching the junction with the ascending colon
Without proper nerve cells, patients experience severe constipation, inflammation, bacterial translocation into the bloodstream, and risk of sepsis and premature death. The current standard of care—pull-through surgery—is invasive, costly, and often associated with significant complications, yet remains non-curative.
Significance of FDA Designations
The FDA's dual designations provide Neurenati with several strategic advantages in developing NEU-001:
The Orphan Drug Designation offers:
- Tax credits for qualified clinical trials
- Exemptions from FDA user fees
- Eligibility for seven years of marketing exclusivity upon approval
The Rare Pediatric Disease Designation may qualify NEU-001 for a Priority Review Voucher upon marketing approval, which could expedite the FDA review process for a future application.
"With a U.S. prevalence of about 100,000 cases and the congenital nature of Hirschsprung disease, the FDA's decision to grant both ODD and RPDD to NEU-001 underscores the strong preclinical proof of concept demonstrated in gold standard animal models," said Dr. Ranger. "This recognition further validates our approach and reinforces our commitment to advancing NEU-001 through clinical development. We believe that NEU-001 has the potential to be a curative therapy for babies diagnosed with Hirschsprung disease, a devastating disease with no approved treatment options."
Development Timeline and Funding
Neurenati is currently conducting an investigational new drug (IND)-enabling preclinical program for NEU-001, with plans to initiate a first-in-human trial by the end of the first half of 2026.
To support this development, the company recently closed an extended seed financing round of $1.7 million led by Genson Capital and Invest Quebec, with additional support from new and existing angel investors. The funding will provide capital to pursue cGMP production of the neurotrophic growth factor, initiate IND-enabling preclinical studies, and expand Neurenati's core team.
Leadership Expansion
As part of its growth strategy, Neurenati has appointed Dr. Meriam Kabbaj, DPharm, PhD, as Chief Development Officer. Dr. Kabbaj will lead the company's overall R&D strategy, including regulatory affairs, preclinical and clinical development of NEU-001.
Dr. Kabbaj brings valuable experience in drug development for rare pediatric diseases. Prior to joining Neurenati, she served as Chief Technology Officer at Genfit S.A., following its acquisition of Versantis, a Switzerland-based company she co-founded and led as Chief Operations Officer.
Looking to the Future
If successful in clinical development, NEU-001 could transform the treatment paradigm for Hirschsprung disease, offering a non-surgical alternative that addresses the root cause of the condition rather than merely managing symptoms.
For the approximately 100,000 patients in the U.S. living with Hirschsprung disease, NEU-001 represents a potential breakthrough that could eliminate the need for invasive surgery and reduce lifelong complications. As Dr. Ranger summarized the company's mission: "We hope for a cure, one child at a time."
