NICE Endorses Rhythm's Imcivree for Rare Genetic Obesity

A groundbreaking treatment targeting obesity and hunger control linked to a genetic cause will soon be accessible through England and Wales' National Health Service (NHS). This development follows the recommendation by the National Institute for Health and Care Excellence (NICE) for the use of Rhythm Pharmaceuticals' Imcivree (setmelanotide) injection in patients with Bardet-Biedl syndrome.

Imcivree has been approved for use in children aged six to 17 who have a confirmed genetic diagnosis of Bardet-Biedl syndrome. Those benefiting from the treatment can continue its use into adulthood. Rhythm Pharmaceuticals, based in the United States, anticipates that Imcivree will be available on the NHS within the next three months. The company is also pursuing approval from the Scottish Medicines Consortium, with a decision expected next year.

Bardet-Biedl syndrome is a rare genetic disorder impacting multiple bodily functions, including vision, the number of digits, and kidney function. A significant aspect of the syndrome is early-onset obesity, largely due to reduced energy requirements and persistent hunger, or hyperphagia. According to the NHS, managing weight is particularly challenging for patients with Bardet-Biedl syndrome because of these factors.

Dr. Philip Beales from the UCL Great Ormond Street Institute of Child Health highlighted the severe impact of hyperphagia, noting that it leads to lifelong, severe obesity affecting many aspects of daily life. Until now, no licensed treatments were available for the obesity and hunger issues caused by Bardet-Biedl syndrome.

Imcivree works by activating melanocortin receptor 4, which enhances the feeling of fullness after eating, thereby reducing weight and body mass index (BMI) as well as hyperphagia. The drug received European approval in 2021 for treating obesity and hunger control linked to deficiencies in three specific genes. In 2022, the UK's Medicines & Healthcare products Regulatory Agency (MHRA) expanded the approval to include patients with Bardet-Biedl syndrome.

Beyond its current approvals, Rhythm Pharmaceuticals has ambitious plans for Imcivree. The company reported promising results from a Phase II trial investigating the drug's effectiveness in treating hypothalamic obesity last year.

In addition to Imcivree, Rhythm Pharmaceuticals expanded its portfolio in January 2024 by licensing LB54640, a clinical-stage weight loss drug from South Korea-based LG Chem, in a deal potentially worth up to $305 million. This acquisition underscores Rhythm's commitment to addressing obesity through advanced treatments and broadening its therapeutic offerings.

As increasing numbers of weight-loss drugs become available, the introduction of Imcivree represents a significant advancement, particularly for those with Bardet-Biedl syndrome. The new treatment offers hope for better managing the severe obesity and constant hunger associated with this genetic condition, marking an important step forward in the field of medical genetics and obesity treatment.