Vitality Biopharma is seeking orphan drug designation from the U.S. Food and Drug Administration (FDA) for its targeted cannabinoid therapy, VITA-100, for the treatment of pediatric ulcerative colitis.
The FDA grants orphan drug status to promising treatments for rare diseases affecting fewer than 200,000 people per year in the United States.
Upon approval, the designation is expected to expedite and facilitate the clinical development and regulatory review of the treatment. The regulatory status also offers additional incentives, including tax credits for clinical testing, seven years of market exclusivity, fast-tracking for regulatory proceedings, and exemption from prescription drug user fees.
“In young children, ulcerative colitis can often be hyperactive and difficult to control using existing FDA-approved medications,” Robert Brooke, CEO and co-founder of Vitality Biopharma, said in a press release.
VITA-100 was developed to deliver directly to the gut two of the active ingredients of cannabis plants — cannabidiol and tetrahydrocannabinol (THC) — to reduce localized inflammation and pain. Because the activity of the drug is targeted to the gut, it does not cause the psychotropic effects in the brain that are commonly linked to cannabis.
The new compounds developed by Vitality belong to a new class of treatments known as cannabinoid prodrugs. Based on the company’s development platform, the cannabinoid compounds are combined with sugar molecules into prodrug formulations. This allows the therapeutically active molecules to be directly delivered to the gut with reduced systemic toxicity.
Pre-clinical studies in animal models of colitis showed that cannabis-based therapies can reduce colon damage and prevent weight loss, while significantly improving gastrointestinal health compared to placebo. Another study has also shown that these cannabinoid compounds can help to induce remission in drug-resistant Crohn’s disease cases.
“Given the mounting clinical data documenting use of cannabinoids for treatment of gastrointestinal disorders, we are very excited about the potential of VITA-100 and our cannabosides platform to provide a meaningful impact for these patients, helping them to stabilize their disease and avoid debilitating surgeries,” he added.
Vitality plans to initiate clinical trials to test the safety and efficacy of VITA-100 as a potential treatment for bowel diseases, irritable bowel syndrome, and narcotic bowel syndrome during the first half of 2018.