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FDA Grants ODD and RPDD to NEU-001 for Patients With Hirschsprung Disease
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The treatment is currently undergoing evaluation in an investigational preclinical program and is projected to be in human trials in 2026.
The FDA granted an orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to NEU-001 (Neurenati Therapeutics, Inc.) for the treatment of patients with Hirschsprung disease. The treatment’s purpose is to serve as a less invasive, nonsurgical option for neonatal patients with the disease.1
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NEU-001 is a novel combination therapy that utilizes a neurotrophic growth factor in patients with Hirschsprung disease. It is a first-in-class, once-in-a-lifetime treatment that is administered intrarectally to regenerate the enteric nervous system in newborns with Hirschsprung disease. The treatment aims to restore gastrointestinal (GI) motility after birth, therefore eliminating the need for pull-through surgery, which is the current standard of care. Because this surgery is an invasive and costly non-curative procedure often associated with significant complications, there is a need for other less intensive treatments. Currently, there is an ongoing investigational new drug-enabling preclinical program for NEU-001, with plans to initiate a first-in-human trial by the end of the first half of 2026.1
Hirschsprung disease is a life-threatening congenital GI disorder that is caused by the absence of nerve cells in parts of the lower GI tract. It is diagnosed in approximately 80% of neonatal patients during the first 4 weeks of life, according to Neurenati Therapeutics. The disease is classified into 2 types depending on the length of the aganglionic intestinal segment: short segment, which is the most common and encompasses only the rectosigmoid colon; and long segment, in which the segment extends beyond the sigmoid colon, sometimes reaching the junction with the ascending colon in certain patients. Having this disease can result in severe constipation, inflammation, bacterial translocation into the bloodstream, sepsis, and premature death.2
“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,” Maxime Ranger, PhD, MBA, CEO of Neurenati Therapeutics, explained in a news release. “Our goal is to offer a non-surgical therapy aimed at promoting nerve cell formation to complete the development of the enteric nervous system.”3
The ODD expedites the development of NEU-001 while offering several key benefits, including tax credits for qualified clinical trials, exemptions from FDA user fees, as well as eligibility for 7 years of marketing exclusivity if the drug receives marketing approval. Additionally, the RPDD is granted to therapies targeting serious or life-threatening diseases that primarily affect children. Upon marketing approval, NEU-001 may be eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), which can expedite the FDA review process for a future marketing application offering significant strategic advantages.1
“With a US 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. 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," said Ranger in a news release. “We hope for a cure, 1 child at a time.”1
