NICE backs first treatment for hyperphagia and early onset severe obesity

The UK’s National Institute for Health and Care Excellence (NICE) has issued guidance that recommends Imcivree (setmelanotide) as an option for treating hyperphagia and obesity and controlling hunger caused by pro-opiomelanocortin (POMC) deficiency, including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency in people six years of age and over.

With this recommendation under the Highly Specialised Technologies (HST) pathway, Imcivree will be funded and available for use within 90 days in the National Health Service, said the drug’s developer, Rhythm Pharmaceuticals (Nasdaq: RYTM). It is only recommended if the company provides setmelanotide according to the commercial arrangement, the NICE noted.

POMC and LEPR deficiencies are caused by genetic variants that disrupt signaling of the MC4R pathway, a system in the hypothalamus that regulates hunger, satiety and energy expenditure. Disrupted MC4R signaling causes hyperphagia and early-onset, severe obesity. People living with obesity due to POMC or LEPR deficiency struggle with insatiable hunger, also known as hyperphagia, and extreme obesity beginning at a young age. These diseases severely affect the quality of life of people living with them, as well as that of their families and caregivers, with many reporting a significant psychological burden, which can manifest as poor mental health, low self-esteem or depression. The NICE concluded that obesity caused by POMC or LEPR deficiency is a debilitating condition associated with multiple co-morbidities.