May 28, 2014
Essentialis announced that the U.S. Food and Drug Administration’s Office of Orphan Products Development has granted orphan drug designation to diazoxide choline, the patent protected active in DCCR for the treatment of Prader-Willi syndrome, a rare complex neurobehavioral/metabolic disease for which there is no FDA-approved therapy.
Prader–Willi syndrome afflicts about 1 in 15,000 to 1 in 25,000 individuals, with the US Prader-Willi syndrome population estimated between 12,500 and 21,000. There may be as many as 350,000 Prader–Willi syndrome patients globally. Prader–Willi syndrome is equally frequent in males and females. Clinical features of Prader–Willi syndrome include hypotonia and poor feeding in infancy. Low muscle mass and low resting energy expenditure is present throughout life. Obesity typically begins around age 2 years if the diet is not restricted. Ultimately, the central neurological defect associated with the condition causes Prader–Willi syndrome patients to sense that they are starving and signals them to further conserve energy and to significantly increase their caloric intake. This results in even lower resting energy expenditure, hyperphagia, morbid obesity, and a progression to diabetes. Mental retardation, growth hormone deficiency, behavioral problems and neuroendocrine abnormalities are also characteristic of Prader–Willi syndrome. The death rate among Prader–Willi syndrome patients is about twice that of the general population at all ages.
“We greatly appreciate the FDA’s support of our efforts to evaluate the use of DCCR in the treatment of Prader-Willi syndrome. We are actively recruiting Prader-Willi syndrome patients for a recently initiated clinical study. Initial results from that study should be coming out during Q3 of this year,” said Dr. Neil M. Cowen, President and Chief Scientific Officer of Essentialis.
Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually. Orphan drug designation entitles Essentialis to a seven-year period of marketing exclusivity in the United States for DCCR, if it is approved by the FDA for the treatment of Prader-Willi syndrome, and enables the company to apply for research funding, tax credits for certain research expenses, and a waiver from the FDA’s application user fee.
DCCR is a proprietary crystalline salt of diazoxide in a controlled-release, once-a-day tablet formulation. It is in development for the treatment of Prader-Willi syndrome and hypothalamic obesity. DCCR is covered by multiple issued US and granted EU patents, which provide composition of matter protection until 2028. Essentialis is globally prosecuting claims to the use of pharmaceutical formulations of KATP channel activators including diazoxide and DCCR in the treatment of Prader-Willi syndrome. Essentialis has evaluated DCCR in more than 200 subjects in multiple double-blind, placebo-controlled studies.
About Essentialis, Inc.
Essentialis is a Carlsbad-based pharmaceutical company focused on the development of breakthrough medicines targeted to the ATP-sensitive potassium channel, a metabolically regulated membrane protein whose modulation has potential to treat and prevent a wide range of metabolic, CNS and cardiovascular diseases. For more information visit http://essentialistherapeutics.com/.