Prader-Willi syndrome—a rare and complex genetic condition—affects 1 in every 20,000 to 30,000 people. It causes a range of behavioral, developmental, and intellectual difficulties, but one of the most excruciating symptoms is hyperphagia, or feeling hungry all the time.
“It's this insatiable desire to eat,” says Anish Bhatnagar, CEO of Soleno Therapeutics, a biopharmaceutical company focused on rare diseases. “It takes over their lives, and the only thing you can do is restrict their access to food: lock the refrigerator and pantries. But their brain continues to tell them they're starving, and they tend to develop aggressive, destructive behaviors.” That upends life not only for the person with Prader-Willi syndrome, but for their entire family.
Until recently, there were no treatments for this condition. In March, however, the U.S. Food and Drug Administration approved VYKAT XR (diazoxide choline), a daily tablet that works by regulating the neural pathways associated with a sense of fullness. According to one 10-week trial, adolescents and adults who took VYKAT XR—which has been studied for more than five years—experienced improvements in behaviors like bargaining and manipulating for food, sneaking food, or waking up at night to look for food. And a 13-week study concluded that participants lost body fat and gained lean body mass when they took the drug.
“There appears to be, over time, a progressive improvement in hyperphagia, to the extent that these patients can live much more normal lives,” Bhatnagar says. Young people are sometimes able to go to college, for example, which they previously weren’t able to do because of their constant need to be monitored.