Emerging treatments

Diazoxide choline

Diazoxide choline controlled-release (DCCR) is currently under review by the US Food and Drug Administration (FDA) for management of hyperphagia. DCCR is a K-ATP channel agonist, which may reduce hyperphagia in PWS by down-regulating insulin secretion from pancreatic beta cells and secretion of neuropeptide Y, and activating K-ATP channels in adipose tissue. A double blind, placebo-controlled study that compared DCCR to placebo did not meet its primary endpoint of change from baseline in hyperphagia over a 13 week period, measured by hyperphagia questionnaire scores.[61]​ However, it did show significant improvement in hyperphagia for patients with severe hyperphagia. There was also significant improvement in physician-assessed Clinical Global Impression of Improvement score and reduction of body fat mass.[61] Data from an ongoing trial where patients receive DCCR for 6 months have shown continued improvement in hyperphagia scores after the initial 13 weeks of treatment, as well as improvements in body composition (i.e., improved lean body mass and lean/fat mass ratio) and many of the characteristic behavioural issues in PWS (e.g., repetitive questioning, anxiety, compulsive behaviours, skin picking).[61]

Other drugs

Other drugs being investigated for use in patients with PWS include the synthetic oxytocin analogue carbetocin for management of hyperphagia and pitolisant (a H3 antagonist/inverse agonist) for treatment of excessive daytime somnolence, narcolepsy, and cataplexy.[62][63]​​​[64]

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