The National Institute for Health and Care Excellence (NICE) has recommended Rhythm Pharmaceuticals’ Imcivree (setmelanotide) as a treatment for hyperphagia (insatiable hunger) and early onset severe obesity.
The recommendation follows positive results from clinical trials suggesting that Imcivree may reduce weight and body mass index (BMI) in people with obesity caused by POMC and LEPR deficiencies, with an improvement in hunger and quality of life also evidenced as a result of the treatment.
POMC and LEPR deficiencies are rare genetic disorders of obesity caused by genetic variants that disrupt signalling of the MC4R pathway, a system in the structure of the brain that regulates hunger, satiety and energy expenditure.
Those living with POMC and LEPR deficiencies are affected by hyperphagia and extreme obesity from a young age, severely affecting their quality of life. Many patients also report a significant psychological burden, manifesting as poor mental health, low self-esteem or depression.
“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 the National Institute for Health and Care Research Cambridge Biomedical Research Centre.
NICE concluded that obesity caused by POMC or LEPR deficiency is a ‘debilitating condition associated with multiple comorbidities’, with current management focusing on dietary restrictions and lifestyle changes, including exercise.
Commenting on the recommendation, David Meeker, chair, president and chief executive officer of Rhyme, said: “We are delighted by this positive NICE recommendation, which makes available the first therapeutic option for patients with POMC or LEPR deficiency in England and Wales.
“We are grateful to NICE for its close partnership throughout this review process and look forward to continued collaboration with Health Technology Assessment bodies and payers across Europe as we execute on our country-by-country strategy to achieve market access for Imcivree.”
The guidance specifically recommends Imcivree for treating obesity and controlling hunger caused by pro-opiomelanocortin (POMC) deficiency, including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency in patients six years of age and older.
With the recommendation under the Highly Specialised Technologies (HST) pathway, Imcivree will be funded and available for use within 90 days in the NHS.