Rhythm Pharmaceuticals Receives Positive Recommendation from NICE for ▼IMCIVREE (setmelanotide) for Treatment of Obesity and Hyperphagia in Patients with Bardet-Biedl Syndrome

IMCIVREE expected to be funded and available for use in England and Wales within three months through the National Health Service in specialist centres

BOSTON, May 22, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced the National Institute for Health and Care Excellence (NICE) has issued guidance that recommends IMCIVREE (setmelanotide) as an option for treating obesity and the control of hunger (hyperphagia) in genetically confirmed Bardet-Biedl syndrome (BBS) in people 6 years of age and over, if they are between 6 and 17 years of age when treatment starts. Patients may remain on reimbursed setmelanotide as adults whilst they continue to benefit from therapy.

“Access to IMCIVREE in England and Wales is an important milestone in our global efforts to bring this medicine to patients and families with BBS,” said Yann Mazabraud, Executive Vice President, Head of International at Rhythm Pharmaceuticals. “We are pleased with this positive recommendation from NICE, made possible by the collaboration and support of advocacy leaders at BBS U.K. and clinical experts, and engaging and productive discussions with NICE.”

BBS is a rare genetic disease with an estimated prevalence of approximately 900 patients in England and Wales. People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. Results from clinical trials suggest that setmelanotide may reduce hyperphagia, weight and body mass index (BMI) in people aged 6 years and over. The most common adverse events are skin hyperpigmentation, injection site reactions, nausea and headache.

“BBS is a debilitating rare disease that severely affects quality of life of patients and their families,” said Philip Beales, M.D., UCL Great Ormond Street Institute of Child Health. “Hyperphagia – the feeling of extreme hunger that stays with patients all the time – leads to early-onset, life-long, severe obesity that affects many aspects of daily living. Until now there have been no licensed treatments for obesity and hyperphagia caused by BBS.”

The final NICE recommendation is aligned to the European Marketing Authorization (EMA) and U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) approval. With this recommendation under the Highly Specialised Technologies (HST) pathway, IMCIVREE is expected to be funded and available for use within three months in the National Health Service covering England and Wales, and Northern Ireland is expected to adopt NICE guidance. Rhythm is moving ahead with submission to the Scottish Medicines Consortium with a decision expected in 2025.