FDA Orphan Drug Designation
TNX-2900, Tonix’s proprietary potentiated intranasal oxytocin is in the pre-Investigational New Drug (IND) stage as a candidate for the treatment of Prader-Willi syndrome (PWS) and non-organic failure to thrive disease. TNX-2900 has been granted Orphan Drug Designation (ODD) in the US by the FDA for the treatment of PWS.
Prader-Willi syndrome is the most common genetic cause of life-threatening childhood obesity1 and results in lack of suckling in infants and, in children and adults, severe hyperphagia, an overriding physiological drive to eat, leading to severe obesity and other complications associated with significant mortality. There is no approved treatment for either the suckling deficit in babies or the obesity and hyperphagia in older children associated with Prader-Willi syndrome. PWS is a rare disease occurring in approximately one in 15,000 births. Intranasal oxytocin has been shown to improve suckling in newborn animals but also suppresses feeding behaviors in adult animal models.
Tonix’s patented potentiated oxytocin formulation is believed to increase specificity for oxytocin receptors relative to vasopressin receptors as well as to enhance the potency of oxytocin.