FDA Grants Orphan and Pediatric Designations to NEU-001 for Hirschsprung’s Disease
Neurenati Therapeutics Inc., a biotechnology company dedicated to pediatric rare diseases, is excited to announce that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug and Pediatric Rare Disease designations to NEU-001 for the treatment of Hirschsprung disease (HD).
Neurenati Therapeutics Inc., a biotechnology company focused on developing innovative treatments for pediatric rare diseases, has made significant strides with its lead program, NEU-001. The company is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted NEU-001 both Orphan Drug and Pediatric Rare Disease designations for the treatment of Hirschsprung disease (HD). This marks a major step forward for the company in its pursuit of transforming care for newborns with HD, a congenital disorder that significantly impacts gastrointestinal function.
What is Hirschsprung Disease and NEU-001?
Hirschsprung disease (HD) is a life-threatening, congenital condition where parts of the gastrointestinal tract lack nerve cells, causing severe digestive issues and preventing normal bowel movements. Newborns diagnosed with HD often face the need for invasive “pull-through” surgery, which is the current standard of care. However, this surgery is not curative, is costly, and can result in significant complications.
NEU-001 is the first and only intra-rectal treatment specifically designed to regenerate the enteric nervous system in newborns with HD. The goal of NEU-001 is to restore gastrointestinal motility in affected infants, eliminating the need for the invasive and non-curative pull-through surgery. By using a neurotrophic growth factor, NEU-001 targets the underlying cause of the disease, potentially providing a curative treatment option. The therapy is still in the preclinical development phase, with an Investigational New Drug (IND) application expected to be filed soon. First-in-human clinical trials are anticipated by the end of the first half of 2026.
FDA Designations and Benefits
The Orphan Drug and Pediatric Rare Disease designations granted to NEU-001 by the FDA are significant milestones for Neurenati. These designations are designed to encourage the development of therapies for rare diseases, specifically those affecting fewer than 200,000 people in the United States. Orphan drug designation comes with various benefits, including tax credits for clinical trials, a waiver of FDA service fees, and a seven-year period of exclusivity following approval.
Additionally, the Pediatric Rare Disease designation grants NEU-001 a potential advantage through the Rare Pediatric Disease Priority Review Voucher. This voucher can expedite the FDA’s review process for a future marketing authorization application, helping to speed up the delivery of this potentially life-saving treatment to patients in need.
“We are excited and honored to receive both Orphan Drug and Pediatric Rare Disease designations from the FDA,” said Dr. Maxime Ranger, CEO of Neurenati Therapeutics. “This dual designation is a testament to the strength of our preclinical proof of concept, supported by well-established animal models. It validates our approach and strengthens our commitment to advancing NEU-001 toward clinical trials. We believe NEU-001 has the potential to provide a curative solution for newborns with HD, a disease for which no approved treatments currently exist.”
Extended Seed Financing and Key Appointments
Neurenati also announced the successful closing of an extended seed financing round, raising $1.7 million. The financing was led by Genson Capital and Investissement Québec, with the participation of both new and existing angel investors. This funding will support the production of the neurotrophic growth factor required for NEU-001 under Good Manufacturing Practices (GMP), allowing the company to continue advancing its preclinical development and work toward submitting an IND application. Additionally, Neurenati plans to expand its team to further support these efforts.
To further strengthen its leadership team, Neurenati has appointed Dr. Meriam Kabbaj, DPharm, PhD, as the new Chief Development Officer. Dr. Kabbaj brings extensive experience in pharmaceutical development, particularly in the pediatric rare diseases space. She previously served as Chief Technology Officer at Genfit SA and played a pivotal role in the acquisition of Versantis AG, a Swiss company she co-founded and led as Chief Operating Officer. Dr. Kabbaj’s deep expertise in regulatory affairs, preclinical and clinical development will be instrumental in driving the global R&D strategies for NEU-001.
About Neurenati Therapeutics
Neurenati Therapeutics is a biotechnology company based in Quebec, Canada, with a primary focus on developing innovative therapies for rare diseases. The company’s lead program, NEU-001, aims to offer a breakthrough treatment for Hirschsprung disease, a congenital disorder that severely affects the digestive system. Neurenati is developing a unique combination therapy utilizing a first-generation growth factor to address the underlying causes of HD, thus eliminating the need for surgery and reducing the associated complications.
Neurenati’s mission is to provide transformative, life-changing therapies for pediatric rare diseases, and with NEU-001, the company is positioning itself to make a significant impact in the treatment of HD. Through the strategic use of growth factors and other cutting-edge technologies, Neurenati is poised to help transform the standard of care for children with this devastating condition.
With the support of its investors, partners, and a talented team, Neurenati is committed to advancing NEU-001 through the clinical development pipeline and offering new hope to families affected by Hirschsprung disease.
