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Essentialis granted ODD for Prader-Willi drug

Orphan drug designation entitles Essentialis to a seven-year period of marketing exclusivity in the US for DCCR

Essentialis has announced that the FDA’s Office of Orphan Products Development has granted orphan drug designation (ODD) 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.

"We greatly appreciate the FDA's support of our efforts to evaluate the use of DCCR in the treatment of Prader-Willi syndrome,” said Dr Neil M Cowen, President and Chief Scientific Officer of Essentialis. “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."

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 US 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 has evaluated DCCR in more than 200 subjects in multiple double-blind, placebo-controlled studies.

According to the cmopany's website, "Insulin and leptin coordinately regulate caloric intake and energy expenditure by inhibiting Neuropeptide Y/Agouti Related Protein (NPY/AGRP) neurons and stimulating proopiomelanocortin (POMC) neurons in the hypothalamus.  Deficiencies in the hypothalamus of either insulin or leptin, or resistance to either will lead to dysregulation of appetite and energy expenditure characterized by increased appetite, which may present as hyperphagia, and reduced energy expenditure. Leptin interacting with its receptor in these neurons, or insulin interacting with its receptor triggers a cascade, one effect of which is to open the KATP channel.  Those who have studied it most closely have suggested that the KATP channel may function as the molecular end-point of the pathway following leptin activation of the leptin receptor in these hypothalamic neurons.  Treatment with DCCR can directly open the KATP channel in these neurons offering the potential to overcome hypothalamic resistance to the action of leptin and/or insulin and, thereby, re-establish control over appetite and energy expenditure."

Prader–Willi syndrome afflicts about 1 in 15,000 to 1 in 25,000 individuals, with the Prader-Willi syndrome population in the US estimated between 12,500 and 21,000.  There may be as many as 350,000 Prader–Willi syndrome patients globally. 

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 two 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 and the death rate among patients is about twice that of the general population at all ages.

Essentialis is 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.

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