setmelanotide for treating obesity caused by LEPR or POMC deficiency

Last edited 11/2022 and last reviewed 12/2022

NICE state (1):

  • Setmelanotide is recommended, within its marketing authorisation, as an option for treating obesity and controlling hunger caused by pro-opiomelanocortin (POMC) deficiency, including proprotein convertase subtilisin/kexin type 1 or leptin receptor (LEPR) deficiency in people 6 years and over. It is only recommended if the company provides setmelanotide according to the commercial arrangement

The NICE committee stated:

  • "..POMC and LEPR deficiencies are rare genetic disorders of obesity that severely affect the quality of life of people with them, and their families and carers. They cause early onset, extreme obesity and hyperphagia (characterised by a feeling similar to starvation) and are linked with many chronic conditions. They are also likely to shorten life expectancy. Current management (best supportive care) focuses on dietary restrictions and lifestyle changes, including exercise..Results from clinical trials suggest that setmelanotide may reduce weight and body mass index (BMI) in people with obesity caused by POMC and LEPR deficiencies. Evidence also suggests that hunger and quality of life are improved with setmelanotide. Longer-term evidence suggests there might be an ongoing treatment effect with setmelanotide, but this is uncertain.."

Setmelanotide in Bardet-Biedl syndrome

A randomised controlled trial (n=38) found setmelanotide resulted in significant bodyweight reductions in patients with Bardet-Biedl syndrome (32.3% reached at least 10% bodyweight reduction after 52 weeks of treatment); however, these results were inconclusive in patients with Alstrom syndrome

  • most commonly reported treatment-emergent adverse events were skin hyperpigmentation (23 [61%] of 38) and injection site erythema (18 [48%])

Reference:

Related pages:

obesity