According to Tonix Pharmaceuticals, the FDA has granted Rare Pediatric Disease Designation to TNX-2900 intranasal potentiated oxytocin for the treatment of Prader-Willi syndrome (PWS) in children and adolescents. Tonix licensed TNX-2900 from the French National Institute of Health and Medical Research (Inserm) in February 2021, and the FDA granted orphan drug designation to the nasal spray in 2022. Tonix says that the FDA cleared an IND for TNX-2900 in 2023.
Tonix has also been developing a different intranasal potentiated oxytocin formulation, TNX-1900, for the treatment of migraine, obesity, and social anxiety disorder. The company from The company initially licensed TNX-1900 for the treatment of migraine from Trigemina in June 2020.
Tonix CEO Seth Lederman commented, “The Rare Pediatric Disease Designation is an important regulatory milestone in the development of TNX-2900. With PWS being the most common genetic cause of life-threatening childhood obesity, we are excited that the FDA has recognized this significant unmet need in children and adolescents, particularly for PWS hyperphagia, which currently has no approved treatments. As PWS is a genetic disorder associated with abnormalities of the oxytocin system, Tonix believes TNX-2900’s unique formulation has the potential to improve intranasal oxytocin’s therapeutic action by addressing limitations in efficacy observed at high-dose intranasal oxytocin that is not Mg2+-potentiated.”
In January 2022, Levo Therapeutics received a CRL to its application for LV-101 intranasal carbetocin for the treatment of excessive hunger and anxiety associated with Prader-Willi syndrome. Acadia Pharmaceuticals, which acquired Levo in June 2022, recently announced that it has initiated a Phase 3 trial of that nasal spray, now known as ACP-101, in people with PWS.
Read the Tonix Pharmaceuticals press release.
