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)
“Over 30 million people in the
“AT is a condition where children suffer from the adverse effects of premature aging and face a very limited life expectancy,” said Dr.
About Ataxia Telangiectasia (AT)
Ataxia 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.
There 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
Clinical Research on NRC and AT
To date, NRC has been investigated in two third-party funded, peer-reviewed published clinical trials for the treatment of AT. The first study published in Movement Disorders demonstrated that supplementation with NRC improved AT scores and increased immunoglobulins, or antibodies, in the immune-compromised patients, with AT score improvements reversing once supplementation concluded (Veenhius et al., 2021).
The second phase II two-year long study, also published in Movement Disorders, demonstrated that long-term NRC supplementation increased whole blood NAD+ levels up to fourfold, and improved neuromotor coordination and eye movements in 90% of participants while maintaining biomarkers of stable liver and kidney function, as compared to historical disease progression (Presterud et al., 2023). Both studies reported no serious adverse events, with NRC being generally well-tolerated.
Not associated with ChromaDex’s future NRC IND filing, there are two additional investigator-initiated ongoing registered clinical trials that will examine NR supplementation in AT patients. The first is a continuation of Presterud et al. 2023 and will track AT patients over the course of 8-10 years. Recently registered, the second will be a single-arm open-label clinical trial scheduled to commence this year in
Significance of Orphan Drug Designation (OOD) and Rare Pediatric Disease Designation (RPD)
According to the FDA, there are too few treatments for rare diseases because of high research and development costs, which companies often cannot recoup as a result of small patient populations. To incentivize companies to invest in bringing treatments to market for rare diseases, in 1983,
Related to ODD, the FDA’s Rare Pediatric Disease (RPD) designation, further incentivizes companies to invest in rare childhood diseases by providing a voucher program to applicants approved by
Future Clinical Trials on NRC and AT
With both designations granted,
“We are excited at the thought of embarking on these important future clinical trials for NRC as we are committed to advancing this to provide hope and relief for those suffering from this debilitating disease,” commented Dr.
For additional information on the science supporting NRC and for future updates visit www.chromadex.com.
Forward-Looking Statements:
This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as “expects,” “anticipates,” “intends,” “estimates,” “plans,” “potential,” “possible,” “probable,” “believes,” “seeks,” “may,” “will,” “should,” “could” or the negative of such terms or other similar expressions, and include the statements regarding the potential benefits and development of NRC as a treatment for AT or other diseases, including statements regarding clinical trials and obtaining IND Designation from the FDA. These forward-looking statements are based on the Company’s current expectations and are subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the ability to continue to pursue additional studies, human trials, and to obtain an IND Designation from the FDA; whether the potential benefits of NRC can be further supported; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; our ability to maintain sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; and the risks and uncertainties associated with our business and financial condition. More detailed information about
About
Setting the benchmark as the gold standard in scientific rigor, safety, quality, and transparency,
ChromaDex’s robust patent portfolio protects NR or nicotinamide riboside chloride (NRC) and other NAD+ precursors.
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ChromaDex Media Contact:
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