1.1 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.
Why the committee made these recommendations
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.
There are also other uncertainties in the modelling, particularly around the quality-of-life decrement value associated with severe hyperphagia. Despite the uncertainties, setmelanotide is likely to provide important clinical and psychological benefits for people with the condition and their carers, and value for money within the context of a highly specialised service. So, setmelanotide is recommended.