Neurenati Therapeutics Inc., a biotechnology company dedicated to developing transformative therapies for rare pediatric diseases, is pleased to announce that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to NEU-001, a novel combination therapy leveraging a neurotrophic growth factor for the treatment of Hirschsprung disease (HD).

NEU-001 is a first-in-class, single-cycle, intrarectally administered treatment designed to regenerate the enteric nervous system (ENS) in newborns with HD. Its primary goal is to restore normal gastrointestinal motility shortly after birth, thereby eliminating the need for pull-through surgery, the current standard of care, which is invasive, non-curative, and often associated with high costs and significant post-operative complications.

Neurenati’s IND-enabling preclinical program for NEU-001 is currently underway, with plans to initiate a first-in-human clinical trial by the end of 2026.

The EMA grants Orphan Drug Designation to encourage the development of medicines intended for rare conditions affecting fewer than 5 in 10,000 individuals in the European Union. This designation provides several 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. Maxime Ranger, Chief Executive Officer of Neurenati Therapeutics. “This European designation further validates our scientific approach and strengthens our commitment to advancing NEU-001 through clinical development. 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.”

ABOUT NEURENATI

Neurenati Therapeutics is a Québec-based biotech company dedicated to developing innovative therapies for rare pediatric diseases. Its lead program focuses on Hirschsprung disease (HD), a life-threatening congenital gastrointestinal (GI) disorder caused by the absence of nerve cells in parts of the lower GI tract. Neurenati is advancing a first-in-class combination therapy incorporating a growth factor, aiming to eliminate the need for surgery and minimize associated complications.

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