The U.S. Food and Drug Administration (FDA) has recently awarded Rare Pediatric Disease Designation for TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi Syndrome (PWS).
PWS is a genetic condition caused by missing or non-working genes on chromosome 15. PWS symptoms affect many parts of the body. Infants with PWS have severe hypotonia (low muscle tone), feeding difficulties, and slow growth. Other signs and symptoms often include:
- Short stature
- Hypogonadism
- Developmental delays
- Cognitive impairment
- Distinctive behavioral characteristics such as temper tantrums, stubbornness, and obsessive-compulsive tendencies.
Intranasal potentiated oxytocin is a magnesium-potentiated formula that enhances the potency of oxytocin and increases specificity for oxytocin receptors relative to vasopressin receptors. In 2022, the drug was granted Orphan Drug designation by the FDA and the investigational new drug application was accepted in 2023.
For more information on Prader-Willi syndrome and other rare genetic disorders, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/