EMOTIVE
BOSTON, USA, Monday 18th July, 2022 – Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced that the National Institute for Health and Care Excellence (NICE) has issued guidance that recommends IMCIVREE® (setmelanotide) as an option 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 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.
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.1,2 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.
Alex Potter, a participant in the Rhythm clinical trial and a patient representative in the NICE deliberations, said, “LEPR deficiency casts a shadow over every aspect of life. Hunger permeates every thought, action, and conversation. The continuous search for food and severe weight gain has affected my engagement in normal daily activities, which in turn makes building meaningful relationships challenging. For me, participating in this trial has always been about giving other people the chance to live a more normal life – one I never had. This decision will affect the lives of so many families who, like me, continue to be physically and psychologically impacted by this disease.”
“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, M.D., Ph.D., Professor at the Wellcome-MRC Institute of Metabolic Science and NIHR 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.”
The final NICE recommendation is aligned to the European Marketing Authorization (EMA) and UK Medicines and Healthcare Products Regulatory Agency (MHRA) approval as the only authorised treatment option for these rare genetic diseases of obesity. The most common adverse events were skin hyperpigmentation, injection site reactions, nausea and headache.
“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,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We are grateful to NICE for their 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.”
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