NICE Recommends IMCIVREE® for Bardet-Biedl Syndrome Treatment

Rhythm Pharmaceuticals, Inc., a global commercial-stage biopharmaceutical company, announced that the National Institute for Health and Care Excellence (NICE) has issued guidance endorsing IMCIVREE® (setmelanotide) as a treatment option for obesity and hunger control in genetically confirmed Bardet-Biedl syndrome (BBS) for individuals aged six years and older, provided the treatment commences between the ages of six and seventeen. Patients benefiting from the therapy during childhood may continue to receive it as adults. This decision marks a significant milestone for Rhythm Pharmaceuticals' efforts to make IMCIVREE accessible to patients in England and Wales.

BBS is a rare genetic condition affecting around 900 individuals in England and Wales. It is characterized by insatiable hunger (hyperphagia) and severe obesity starting early in life. Clinical trials have shown that setmelanotide can reduce hyperphagia and decrease weight and body mass index (BMI) in patients aged six and above. Common side effects include skin hyperpigmentation, injection site reactions, nausea, and headaches.

Dr. Philip Beales from the UCL Great Ormond Street Institute of Child Health commented on the debilitating nature of BBS, emphasizing that the disease significantly impacts the quality of life for patients and their families due to the extreme and constant hunger that leads to severe obesity. Until now, there were no licensed treatments available for managing obesity and hyperphagia in BBS patients.

The NICE recommendation aligns with approvals from both the European Marketing Authorization (EMA) and the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA). The decision, part of the Highly Specialised Technologies (HST) pathway, ensures that IMCIVREE will be funded and available for use within three months in the National Health Service (NHS) for England and Wales. Northern Ireland is also expected to adopt this guidance. Rhythm Pharmaceuticals plans to submit IMCIVREE for approval to the Scottish Medicines Consortium, with a decision anticipated in 2025.

Rhythm Pharmaceuticals is dedicated to transforming the lives of patients with rare neuroendocrine diseases. Their lead product, IMCIVREE (setmelanotide), is an MC4R agonist designed to manage hyperphagia and severe obesity. The U.S. Food and Drug Administration (FDA) has approved setmelanotide for chronic weight management in adult and pediatric patients aged six years and older with specific genetic deficiencies confirmed by testing, or with a clinical diagnosis of BBS. Both the European Commission (EC) and the UK’s MHRA have authorized setmelanotide for treating obesity and hunger control in individuals with genetically confirmed BBS or specific genetic deficiencies in adults and children aged six and above.

While setmelanotide has shown promise, it is not indicated for treating obesity due to non-genetic causes or other types of genetic obesity not related to the specific deficiencies targeted by the drug. The therapy has some limitations and risks, including potential skin pigmentation changes, blood pressure alterations, sexual arousal disturbances, and depression. Patients undergoing treatment should be closely monitored for these adverse effects.

Ultimately, the availability of IMCIVREE in the NHS represents a significant advancement for patients with BBS in England and Wales, offering them a new, effective treatment option for managing their condition.