Neurenati Therapeutics Inc. has received Orphan Drug Designation from the European Medicines Agency for NEU-001, a novel combination therapy targeting Hirschsprung disease, a rare congenital gastrointestinal disorder affecting newborns. The Montreal-based biotechnology company announced this regulatory milestone on October 28, 2025, marking a significant step forward in developing the first pharmacological treatment for this life-threatening condition.
Revolutionary Approach to Hirschsprung Disease
NEU-001 represents a first-in-class, single-cycle treatment administered intrarectally to regenerate the enteric nervous system in newborns diagnosed with Hirschsprung disease. The therapy leverages a neurotrophic growth factor designed to restore normal gastrointestinal motility shortly after birth, offering a potential alternative to the current standard of care.
The innovative approach aims to eliminate the need for pull-through surgery, which Dr. Maxime Ranger, Chief Executive Officer of Neurenati Therapeutics, describes as "invasive, non-curative, and often associated with high costs and significant post-operative complications." This surgical intervention currently represents the only treatment option for patients with Hirschsprung disease, a condition caused by the absence of nerve cells in parts of the lower gastrointestinal tract.
Regulatory Recognition and Development Timeline
The EMA's Orphan Drug Designation follows previous recognition from the U.S. FDA, which granted both Orphan Drug and Rare Pediatric Disease Designations to NEU-001. This European designation provides several development incentives, including protocol assistance, 10-year market exclusivity upon marketing authorization, and fee reductions. For orphan medicines intended for pediatric use, an additional two years of market exclusivity may be granted.
"Following the U.S. FDA's Orphan Drug and Rare Pediatric Disease Designations, we are thrilled that the EMA has now also recognized NEU-001's potential to transform the life of patients suffering from Hirschsprung disease, a life-threatening and chronically debilitating condition," said Dr. Ranger. "This European designation further validates our scientific approach and strengthens our commitment to advancing NEU-001 through clinical development."
Clinical Development Progress
Neurenati's IND-enabling preclinical program for NEU-001 is currently underway, with the company planning to initiate a first-in-human clinical trial by the end of 2026. The development timeline reflects the company's systematic approach to advancing this potential breakthrough therapy through regulatory pathways.
The EMA grants Orphan Drug Designation to encourage development of medicines for rare conditions affecting fewer than 5 in 10,000 individuals in the European Union. Hirschsprung disease meets these criteria as a rare congenital disorder with no currently approved pharmacological treatment options.
Addressing Unmet Medical Need
Dr. Ranger emphasized the potential impact of NEU-001, stating, "We believe NEU-001 has the potential to be a curative therapy for newborns diagnosed with HD, a devastating disease with no approved pharmacological treatment options. We hope for a cure, one child at a time."
The company's focus on rare pediatric diseases positions NEU-001 as a potentially transformative therapy for families affected by Hirschsprung disease, offering hope for a non-surgical treatment approach that could significantly improve patient outcomes and quality of life.
