DCCR is a novel, crystalline patent-protected salt of diazoxide formulated as a controlled release once-a- day tablet. Diazoxide free base is approved as a three-times- a-day oral suspension that has been used safely for decades in tens of thousands of patients.
Diazoxide, a K ATP channel agonist or opener, is first line therapy in a range of rare diseases in neonates, children and adults. Over the last 40 years, there have been more than 3800 peer reviewed publications covering in-vitro, animal model, case reports and clinical studies with diazoxide.
Essentialis has assembled an extensive toxicological package on the product which shows a wide margin of safety for therapeutically relevant doses.
The safety profile of diazoxide is well known, with more than 100,000 patient years of use. DCCR has been tested in 8 clinical trials involving nearly 300 randomized patients including more than 210 treated with DCCR. There have been no new safety findings with DCCR that had not previously been observed with diazoxide.
Prader-Willi syndrome is a rare disease. The prevalence in the US is substantially less than 200,000 patients. Diazoxide choline controlled release has received an orphan drug designation from US FDA for the treatment of Prader-Willi syndrome and anticipates being able to obtain a 7-year grant of market exclusivity from the FDA in this indication.