Essentialis receives orphan drug designation for Prader-Willi syndrome treatment
- FDA gave orphan drug designation to Essentialis for DCCR, a proprietary crystalline salt of diazoxide formulated into a once-daily tablet.
- This treatment will be evaluated in clinical trials involving patients with Prader-Willi syndrome.
- There is currently no treatment for Prader-Willi syndrome other than strict control of eating behavior.
There is currently no treatment for Prader-Willi syndrome, a neurobehavioral/metabolic disorder in which patients do not register satiety and can become morbidly obese without constant oversight. Essentialis, which has received orphan drug designation for DCCR, is currently enrolling subjects for a clinical trial evaluating the use of this once-daily tablet-based medication for patients with Prader-Willi syndrome.