Tonix Pharmaceuticals Holding Corp (NASDAQ: TNXP) has announced that the FDA has granted TNX-2900 an Orphan Drug Designation for Prader-Willi syndrome treatment.
Tonix receives Orphan Drug Designation from FDA
CEO Seth Lederman said, “Orphan-Drug Designation by the FDA is an important milestone and further validates our efforts to investigate the utility of TNX-2900 for Prader-Willi syndrome. It underscores the urgent, unmet medical need for patients diagnosed with this disease, and will benefit us as we continue to advance our program.”
Recently the company disclosed a sponsored research contract with Inserm’s subsidiary, Inserm Transfert, and Aix Marseille Universite to evaluate oxytocin in a genetically modified mouse model of Prader-Willi syndrome. Prader-Willi syndrome is a rare genetic disease that results in distinct but associated pathological eating disorders in newborns and adults. Symptoms of the disease include pathological overeating to hyperphagia in adults, often associated with obesity and other problems related to high mortality rates. Notably, the disorder causes a suckling insufficient in neonates that can be corrected by oxytocin. Unfortunately, there is no available drug for Prader-Willi syndrome-related suckling deficiencies in neonates or obesity hyperphagia in older children.
The FDA’s Office of Orphan Drug Products gives orphan status to active ingredients in medicines and biologics that show promise in treating diseases or disorders that affect fewer than 200,000 individuals in the US. Tonix Pharmaceuticals will benefit from orphan drug status, which includes tax subsidies for qualified clinical trials, exemptions from some FDA application expenses, and the possibility of seven years of commercial exclusivity if approved.
TNX-2900 is an intranasal potentiated oxytocin mixture
TNX-2900 is a proprietary intranasal stimulated oxytocin mixture for adults and adolescents developed by Tonix. Tonix’s proprietary potentiated oxytocin formula is thought to boost oxytocin receptor selectivity in comparison to vasopressin receptors while also increasing oxytocin potency. Tonix is also working on TNX-1900, a new intranasal formula and device for preventing chronic migraine and the management of insulin resistance and associated illnesses. Oxytocin is a neurotransmitter in the brain that is produced spontaneously by humans.