Prader-Willi Reminds Us GLP-1 Is Not the Whole Story of Obesity

While much of the public has focused on the remarkable effects of GLP-1 agonists for many people living with obesity, an even more remarkable story has largely escaped notice. In March, FDA approved the first ever treatment for Prader-Willi syndrome. This drug, an extended-release form of diazoxide choline, is transforming the lives of families of this rare syndrome associated with obesity. The insatiable hunger that comes with Prader-Willi can make a normal life impossible without treatment.

Its dramatic effects on their lives is a sharp reminder that GLP-1 agonists are not the whole story of obesity. It comes in more forms than most of us appreciate.

Life-Changing Therapy

“Before the drug, it just felt like a dead end. My child was miserable. Now, we have our son back.” These words from Ali Foley Shenk about her son Dean offer a glimpse of the transformation an effective therapy for Prader-Willi syndrome can bring.

Jennifer Miller is a pediatric endocrinologist at the University of Florida, where she led trials that supported this drug’s approval. For a story in KFF Health News, she told Claire Sibonney that she has seen the lives of her adult patients change dramatically during research on this drug. Sibonney writes:

“Since the drug trial started in 2018, some of her adult patients have begun living independently, getting into college, and starting jobs — milestones that once felt impossible. ‘It opens up their world in so many ways.’”

And yet, Prader-Willi, like other forms of obesity, remains a chronic disease. Diazoxide choline does not cure the disease, it controls it. Other trials of other drugs are underway and may lead to even better outcomes. But research on this rare disease is not easy and has relied upon research funded by the NIH. Such research faces threats from dramatic cuts to federal research funding.

More Ways Than One

Obesity is such a common problem and Prader-Willi syndrome is so rare. But it reminds us that obesity has many causes and very likely will require many different therapeutic options.

Diazoxide choline is a potent activator of adenosine triphosphate–sensitive potassium. In the brain, it seems to calm some of the neurons that trigger the extreme hunger that people with Prader-Willi experience. It is a potent drug with significant side effects, but its benefits generally outweigh its risks.

The quiet success of diazoxide choline offers a striking contrast to the loud success of GLP-1 agonists. Both bring value and both work in very different ways. But together they are sparking even more curiosity in different forms of obesity and different pathways for treating them.

We are only barely scratching the surface

Click here for excellent reporting by Sibonney. To read about the approval of Vykat XR (diazoxide choline), click here. For the pivotal trial results with this drug, click here.

The Story of St. Nicholas, Giving Dowry to Three Poor Girls, painting by Fra Angelico / WikiArt

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June 17, 2025