FDA Grants Rare Pediatric Disease Designation to ChromaDex Treatment for AT

Rare Daily Staff

The U.S. Food and Drug Administration granted Orphan Drug and Rare Pediatric Disease designations to ChromaDex for nicotinamide riboside chloride for the treatment of ataxia telangiectasia.

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.

“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.”

To date, nicotinamide riboside chloride (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.

The second phase 2 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 percent of participants while maintaining biomarkers of stable liver and kidney function, as compared to historical disease progression. Both studies reported no serious adverse events, with NRC being generally well-tolerated.

ChromaDex plans to conduct additional studies on NRC in anticipation of filing for an Investigational New Drug Application for the treatment of AT.

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, Congress passed the Orphan Drug Act, which makes Orphan Drug designation candidates eligible for tax credits, waives their user fees, and may provide a period of exclusivity should the orphan drug be subsequently approved by FDA.

The FDA’s Rare Pediatric Disease designation further incentivizes companies to invest in rare childhood diseases by providing a voucher program to applicants approved by September 30th, 2024. Through this program, companies with RPD designation that ultimately obtain successful drug approval for a rare pediatric disease are provided a voucher, which can be used to expedite the FDA review of another drug candidate or sold to other companies.