TNX-2900 to treat Prader-Willi syndrome granted Rare Pediatric Disease designation

Intranasal potentiated oxytocin to treat Prader-Willi syndrome granted Rare Pediatric Disease designation | Image Credit: © luchschenF - © luchschenF - stock.adobe.com.

The FDA has granted Rare Pediatric Disease Designation to intranasal potentiated oxytocin (TNX-2900; Tonix Pharmaceuticals) to treat Prader-Willi syndrome (PWS) in children and adolescents, according to a press release from Tonix Pharmaceuticals.¹

The proprietary magnesium (Mg2+)-potentiated formulation of intranasal oxytocin was previously granted Orphan Drug designation by the federal agency in 2022 for the treatment of PWS. The investigational new drug application was cleared by the FDA in 2023.¹

PWS affects males and females of all races and ethnicities equally, and is recognized as the most common genetic cause of life-threatening childhood obesity. The syndrome results from the absence of expression of a group of genes on the paternally acquired chromosome 15, according to Tonix.¹

In newborns, a hallmark of PWS is lack of suckling. For children and adolescents, the hallmark is severe hyperphagia, "an overriding physiological drive to eat, leading to severe obesity and other complications associated with significant mortality."¹

The average age of death in PWS was 22.1 years following a systematic review of the morbidity and mortality as a consequence of hyperphagia in PWS.¹

Currently, there are no approved medications to treat hyperphagia in children and adolescents with PWS, or to treat poor feeding in newborns.¹

According to Tonix, the following studies demonstrated evidence for improvement in PWS-related symptoms/behaviors, reported evidence for improvement in hyperphagia, or improvement in sucking in infants:¹

• The effects of oxytocin on eating behavior and metabolism in humans²
• Oxytocin treatment in children with Prader-Willi syndrome: A double-blind, placebo-controlled, crossover study³
• The Use of Oxytocin to Improve Feeding and Social Skills in Infants With Prader-Willi Syndrome⁴
• Oxytocin in young children with Prader-Willi syndrome: Results of a randomized, double-blind, placebo-controlled, crossover trial investigating 3 months of oxytocin⁵

References:

1. Tonix Pharmaceuticals receives Rare Pediatric Disease Designation from the FDA to TNX-2900 for the treatment of Prader-Willi Syndrome. Tonix Pharmaceuticals. Press release. March 25, 2024. Accessed March 25, 2024. https://ir.tonixpharma.com/news-events/press-releases/detail/1465/tonix-pharmaceuticals-receives-rare-pediatric-disease

2. Lawson EA. The effects of oxytocin on eating behaviour and metabolism in humans. Nat Rev Endocrinol. 2017 Dec;13(12):700-709. doi: 10.1038/nrendo.2017.115. Epub 2017 Sep 29. PMID: 28960210; PMCID: PMC5868755.

3. Miller JL, Tamura R, Butler MG, et al. Oxytocin treatment in children with Prader-Willi syndrome: A double-blind, placebo-controlled, crossover study. Am J Med Genet A. 2017;173(5):1243-1250. doi:10.1002/ajmg.a.38160

4. Tauber M, Boulanouar K, Diene G, et al. The Use of Oxytocin to Improve Feeding and Social Skills in Infants With Prader-Willi Syndrome. Pediatrics. 2017;139(2):e20162976. doi:10.1542/peds.2016-2976

5. Damen L., Grootjen L.N., Juriaans A.F., et al. Oxytocin in young children with Prader-Willi syndrome: Results of a randomized, double-blind, placebo-controlled, crossover trial investigating 3 months of oxytocin. Clin Endocrinol, 94: 774-785. https://doi.org/10.1111/cen.14387