Niagen Bioscience Receives Exclusive U.S. FDA Rare Pediatric Disease (RPD) Designation and European Medicines Agency Orphan Medicinal Product Designation (OMPD) for NB4168 for the Treatment of Ataxia Telangiectasia (A-T)

About this update from Niagen Bioscience, Inc.

Regulatory recognition in the United States and Europe supports the development of NB4168, a novel small molecule candidate for a rare pediatric disease with no approved treatments LOS ANGELES, July 13, 2026 --( BUSINESS WIRE )-- Niagen Bioscience, Inc. (NASDAQ: NAGE), the global authority on NAD+ (nicotinamide adenine dinucleotide) with a focus on the science of healthy aging, today announced that the U.S. Food & Drug Administration (FDA) granted Rare Pediatric Disease (RPD) Designation for its proprietary lead small molecule drug candidate NB4168 for the treatment of Ataxia Telangiectasia (A-T). NB4168 is an oral small molecule therapy engineered to deliver substantially greater nicotinamide riboside (NR) exposure than conventional NR while maintaining a differentiated pharmacokinetic and safety profile. In addition, the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation (OMPD) to NB4168 for the treatment of A-T, providing regulatory recognition in the European Union and further supporting the Company's plans to advance the program globally. The FDA granted RPD Designation based on its determination that A-T is a serious and life-threatening disease that primarily affects individuals from birth through adolescence and meets the statutory definition of a rare disease. The EMA's Committee for Orphan Medicinal Products similarly concluded that NB4168 met the criteria for orphan designation for the treatment of A-T. Together, these regulatory designations recognize the significant unmet medical need in A-T and provide development incentives intended to support and accelerate the advancement of promising therapies for rare diseases. NB4168 is the first investigational therapeutic candidate to emerge from Niagen Bioscience's recently announced wholly owned subsidiary focused on developing therapies for rare genetic diseases and age-related disorders, NAD Pharmaceuticals Corp . NB4168 is designed to have significantly higher bioavailability and increase NAD+, a coenzyme essential for DNA repair, mitochondrial function, cellular energy production, and stress responses—biological pathways disrupted in A-T. As A-T is categorized as a rare genetic premature aging disease, NB4168 may translate to other age-related diseases. "Receiving RPD Designation from the U.S. FDA and OMPD from the EMA represents meaningful regulatory validation of NB4168 and our strategy to develop therapies for patients with serious rare diseases," said Rob Fried, CEO of Niagen Bioscience. "These milestones strengthen our path toward clinical development and reinforce the opportunity to extend our leadership in NAD+ science into regulated medicines."

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