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ChromaDex Receives Exclusive U.S. FDA Orphan Drug Designation (ODD) and Rare Pediatric (RPD) Disease Designation for Nicotinamide Riboside Chloride (NRC) for the Treatment of Ataxia Telangiectasia (AT)
ChromaDex plans to conduct additional studies on NRC in anticipation of filing for an Investigational New Drug Application (IND) for the treatment of AT LOS
About this update from Niagen Bioscience, Inc.
[{"type":"text","content":"\nChromaDex plans to conduct additional studies on NRC in anticipation of filing for an Investigational New Drug Application (IND) for the treatment of AT\n\n\n LOS ANGELES--(BUSINESS WIRE)--\nChromaDex Corp. (NASDAQ:CDXC), the global authority on nicotinamide adenine dinucleotide (NAD+) and healthy aging research, today announced that the U.S. Food & Drug Administration (FDA) granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) Designation for NRC, the company’s product candidate for the treatment of Ataxia Telangiectasia (AT). Plans are underway to file an Investigational New Drug (IND) application with the U.S. FDA in anticipation of conducting human clinical trials, which will be guided by Dr. Vilhelm (Will) Bohr, Prof., University of Copenhagen and Scientific Advisor to ChromaDex.\n\n\n“Over 30 million people in the U.S. are impacted by more than 7,000 rare diseases, many of which are life-threatening and lack effective treatments,” said Rob Fried, CEO of ChromaDex. “We believe NRC has potential as a treatment for AT.”\n\n\n“AT is a condition where children suffer from the adverse effects of premature aging and face a very limited life expectancy,” said Dr. Andrew Shao, ChromaDex Senior Vice President of Global Scientific & Regulatory Affairs. “We are excited to continue supporting the AT community and eagerly anticipate the results from future research.”\n\n\nAbout Ataxia Telangiectasia (AT)\n\n\nAtaxia Telangiectasia (AT) is a rare, progressive disease that typically presents in early childhood and is characterized by neurological and immunological symptoms. Those with AT often exhibit an unsteady gait (ataxia), impaired coordination of eye movements (oculomotor apraxia), and involuntary movements (choreoathetosis). AT leads to cerebellar degeneration and many affected children become wheelchair-dependent. Currently, there is no cure or FDA-approved treatment to slow the progression of AT, with the average life expectancy being around 25 years for those diagnosed in childhood.\n\n\nThere are many types of ataxia. Another form of ataxia is Friedreich’s ataxia (FA), which is being addressed by Biogen and Larimar Therapeutics. FA impacts 1 in 50,000 people in the U.S. (NIH). Ataxia Telangiectasia (AT) impacts roughly 1 in 40,000 people in the U.S. (Riboldi et al., 2023; Tieve et al., 2015).\n\n\nClinical Re...