Rare Metabolic Disease That Leads to Childhood Obesity Gets Its First FDA-Approved Drug

MedCity News March 27, 2025
Frank Vinluan

Soleno Therapeutics’ Vykat XR is now FDA approved for treating hyperphagia, or excessive hunger, in patients with Prader-Willi syndrome. The once-daily pill is the first approved therapy for this rare disease, a leading cause of childhood obesity.

Obesity has many drivers, but for those with a particular rare metabolic disorder, the root cause is genetic. These patients develop ravenous hunger unsatisfied by any amount of food. The constant eating and food-seeking behavior leads families and caregivers to restrict food access by locking refrigerators, pantries, even trash cans.

This rare disease, Prader-Willi syndrome, has been described in medical literature for nearly 70 years. But even as medical understanding of the disorder grew, efforts to develop a drug to treat it have...

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