The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Jotrol, a potential new therapy for Friedreich’s ataxia (FA), says the medication’s developer, Jupiter Orphan Therapeutics.
Rosén said his expectation follows an earlier Phase 2 trial of resveratrol, a powerful natural antioxidant, conducted by Jupiter’s partner, Murdoch Children’s Research Institute of Melbourne, Australia. The study “generated very encouraging results, where four out of five very important endpoints were met,” he said, adding that these results are likely to be repeated “through a larger placebo-controlled study utilizing Jotrol to avoid gastrointestinal (GI) tolerability issues.”
Rosén observed that positive results in this new study “will hopefully lead to market approval in several territories” including the United States.
The FDA grants orphan drug status to therapies that may treat, diagnose or prevent diseases affecting fewer than 200,000 Americans. This regulatory step gives pharmaceutical companies benefits such as tax credits for clinical testing, seven years of market exclusivity, exemption from prescription drug user fees and protocol assistance from the FDA.
FA, which affects about one in 50,000 Americans, is a rare inherited disease with no approved treatments. It usually starts in childhood and leads to progressive impaired muscle coordination. FA is caused by a mutation in the FXN gene, leading to deficient levels of the protein frataxin.
Studies have shown that resveratrol-based formulations increase frataxin levels, thereby improving the function of mitochondria, the cellular organelles responsible for respiration and energy production. Jotrol is a unique formulation of trans-resveratrol — one of the two possible forms of resveratrol — made by Jupiter. The product is designed to deliver high levels of resveratrol in blood plasma that are needed for therapeutic effects.
However, earlier work showed that these high doses caused severe adverse GI effects. Based on successful pre-clinical results, the Florida-based company expects Jotrol to deliver the required quantities of resveratrol to treat FA without those adverse side effects.