Prader-Willi Syndrome

Once-daily liraglutide was not associated with greater weight loss for youths with obesity and Prader-Willi syndrome compared with placebo, according to a study published in The Journal of Clinical Endocrinology & Metabolism.

Prader-Willi syndrome can delay the diagnosis of hereditary angioedema, which already can be difficult to identify, according to a case report published in Annals of Allergy, Asthma & Immunology.

The FDA’s Division of Psychiatry has rejected a new drug application for treating hyperphagia, anxiousness and distress associated with Prader-Willi syndrome, according to a press release.

The FDA granted orphan drug designation for an investigational combination therapy for the treatment of hypothalamic obesity, according to an industry press release.

In a recent interview, Alice C. Levine, MD, professor of medicine, endocrinology, diabetes and bone disease at the Icahn School of Medicine at Mount Sinai, New York, spoke with Healio about how antiandrogen treatments could potentially improve — or worsen — COVID-19 outcomes. It was the top story in endocrinology last week.

Prader-Willi syndrome, a neurodevelopmental disorder that occurs in about 1 in 15,000 births, is the most common genetic cause of life-threatening childhood obesity, which is just one of its complex symptoms. Still, it has proved extremely difficult to treat. Fortunately for the Prader-Willi syndrome community, researchers continue to test potential therapies to find a comprehensive treatment or a way to curb the most severe symptoms.

Endocrine Today has compiled a list of recent FDA news stories relating to endocrine practice. Drugs under consideration included those for thyroid eye disease, diabetes, hypertriglyceridemia, Prader-Willi syndrome and nonalcoholic steatohepatitis.

The FDA this week granted fast track designation for intranasal carbetocin, an oxytocin analogue, for the treatment of Prader-Willi syndrome, according to a press release from Levo Therapeutics.

A small study suggests that bariatric surgery did not result in long-term, sustainable weight loss or a reduction in comorbidities for adults with Prader-Willi syndrome despite early weight loss, according to findings published in Pediatric Obesity.

Rhythm Pharmaceuticals announced positive results from two phase 3 clinical trials evaluating setmelanotide, a melanocortin-4 receptor agonist for the treatment of rare congenital forms of obesity, according to a company press release.