FDA Grants Orphan & Rare Pediatric Status to NEU-001 for Hirschsprung Disease
Neurenati Therapeutics Inc., a biotechnology company specializing in rare pediatric diseases, has announced a significant regulatory milestone. The U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to NEU-001, the company’s novel combination therapy utilizing a neurotrophic growth factor, for the treatment of Hirschsprung disease (HD). This designation underscores the potential of NEU-001 as a transformative therapeutic approach for newborns diagnosed with this serious congenital disorder.
Hirschsprung disease is a rare developmental disorder affecting the enteric nervous system (ENS), the network of neurons responsible for coordinating intestinal motility. Infants born with HD lack the necessary nerve cells in the lower gastrointestinal tract, leading to severe constipation, bowel obstruction, and life-threatening complications if left untreated. Currently, the standard treatment involves pull-through surgery, a complex and invasive procedure aimed at removing the affected segment of the intestine. However, this surgery does not restore normal gut function and is associated with long-term complications such as incontinence, enterocolitis, and poor quality of life.
NEU-001 represents a paradigm shift in the treatment of Hirschsprung disease. Unlike surgical intervention, NEU-001 is designed as a first-in-class, once-in-a-lifetime therapy administered intrarectally to regenerate the enteric nervous system in newborns. By targeting the underlying neurological deficiency, NEU-001 aims to restore gastrointestinal motility, potentially eliminating the need for pull-through surgery altogether. This novel approach holds promise for transforming patient outcomes by addressing the root cause of the disease rather than merely managing its symptoms.
Neurenati is actively conducting IND-enabling preclinical studies for NEU-001, with plans to initiate a first-in-human clinical trial by the second half of 2026. Preclinical data generated thus far have demonstrated strong proof of concept in gold-standard animal models, further supporting the therapy’s potential efficacy and safety. The FDA’s dual designation of ODD and RPDD highlights the importance of this innovative approach in addressing a high unmet medical need.
The FDA’s Orphan Drug Designation is granted to investigational therapies intended for the treatment of rare diseases affecting fewer than 200,000 individuals in the U.S. This designation provides several key incentives to support drug development, including tax credits for qualified clinical trials, exemption from FDA user fees, and eligibility for seven years of marketing exclusivity upon approval. These benefits help facilitate the development of novel treatments for conditions like Hirschsprung disease, which otherwise may not attract sufficient investment due to the limited patient population.
Similarly, the Rare Pediatric Disease Designation is awarded to therapies targeting serious or life-threatening diseases that primarily affect children. One of the most significant advantages of RPDD is its potential eligibility for a Rare Pediatric Disease Priority Review Voucher (PRV) upon FDA approval. This voucher allows the holder to expedite the review process for a future marketing application, providing a strategic advantage in bringing innovative therapies to market more efficiently. The PRV can also be sold or transferred, offering financial benefits that can further support the company’s research and development initiatives.
“With an estimated prevalence of approximately 100,000 cases in the U.S., Hirschsprung disease remains a serious and often debilitating condition with no approved pharmacological treatments,” stated Dr. Maxime Ranger, CEO of Neurenati Therapeutics. “The FDA’s decision to grant both Orphan Drug and Rare Pediatric Disease Designations to NEU-001 underscores the strength of our preclinical data and validates our commitment to developing a potentially curative therapy for babies diagnosed with this devastating disease. We hope for a cure, one child at a time.”
Beyond its regulatory advancements, Neurenati has also achieved critical financial and organizational milestones to accelerate NEU-001’s development. The company recently closed an extended seed financing round, securing $1.7 million in funding. This round was led by Genson Capital and Invest Quebec, which participated through its Impulsion PME program, a government-mandated initiative supporting promising startups. Additional contributions came from new and existing angel investors. The infusion of capital will be instrumental in advancing the company’s core objectives, including the Good Manufacturing Practice (cGMP) production of the neurotrophic growth factor, completion of IND-enabling preclinical studies, and expansion of the company’s core scientific and operational team.
In alignment with its strategic growth, Neurenati has appointed Dr. Meriam Kabbaj, DPharm, PhD, as its new Chief Development Officer. Dr. Kabbaj brings a wealth of experience in drug development, with a specialized focus on rare pediatric diseases. She will oversee the company’s research and development strategy, including regulatory affairs and the clinical advancement of NEU-001. Prior to joining Neurenati, Dr. Kabbaj served as Chief Technology Officer at Genfit S.A., following its acquisition of Versantis, a Swiss-based biotechnology company she co-founded and led as Chief Operations Officer. She holds a Doctor of Pharmacy (DPharm) from the University of Geneva and a PhD in Pharmacology from the Université de Montréal.
Dr. Kabbaj’s expertise will be pivotal in guiding Neurenati through the critical transition from preclinical to clinical development. Her leadership will ensure that NEU-001 progresses through regulatory milestones efficiently while maintaining the highest scientific and ethical standards in drug development. Her appointment reflects Neurenati’s commitment to assembling a world-class team dedicated to pioneering breakthrough treatments for rare pediatric diseases.
Looking ahead, Neurenati remains steadfast in its mission to advance NEU-001 through clinical trials with the ultimate goal of bringing the first pharmacological treatment for Hirschsprung disease to market. The company’s continued progress in securing regulatory designations, attracting strategic investments, and strengthening its leadership team positions it well for future success.
With FDA support, promising preclinical results, and strong financial backing, Neurenati is poised to make a significant impact in the field of pediatric rare diseases. As the company moves forward, it remains dedicated to its vision of delivering life-changing therapies to the patients and families who need them most. Through innovation, collaboration, and perseverance, Neurenati aims to redefine the treatment landscape for Hirschsprung disease and set a new standard of care for generations to come.
