Rhythm Pharmaceuticals has announced that the National Institute for Health and Care Excellence (NICE) has issued guidance that recommends Imcivree – also known as setmelanotide – as an option for treating obesity and controlling hunger caused by pro-opiomelanocortin (POMC) deficiency. It also involves patients with the leptin receptor (LEPR) deficiency in people six years of age and over.
The most common adverse events were skin hyperpigmentation, injection site reactions, nausea and headache.
The treatment has been developed for people aged six years and over, is provided under the Highly Specialised Technologies (HST) pathway and will be available for use within 90 days across the NHS.
POMC and LEPR deficiencies are caused by genetic variants that disrupt signalling of the melanocortin-4 receptor (MC4R) pathway – a system that regulates hunger, satiety and energy expenditure. Disrupted MC4R signalling 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.
NICE concluded that obesity caused by POMC or LEPR deficiency is a debilitating condition associated with multiple comorbidities.
“The effects of rare MC4R pathway diseases, including POMC and LEPR deficiency, go far beyond a patients’ weight and hunger, severely affecting their ability to maintain a normal quality of life beginning in childhood,” said Sadaf Farooqi, Professor at the Wellcome-MRC Institute of Metabolic Science and National Institute for Health and Care Research, Cambridge Biomedical Research Centre.
“In addition, caring for a person with one of these conditions can be physically and mentally draining for families who are often stigmatized in their communities. This NICE recommendation reflects the value of Imcivree and it is welcome news for clinicians and eligible patients who will soon have access to the first and only therapy to address the underlying cause of obesity and hunger in POMC and LEPR deficiencies,” he added.
The final NICE recommendation is aligned to the European Marketing Authorization and UK Medicines and Healthcare Products Regulatory Agency approval as the only authorised treatment option for these rare genetic diseases of obesity.