From genes to care: the expanding therapeutic horizon in rare genetic obesities

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Abstract Context Advances in our understanding of the molecular causes of rare genetic obesities, particularly those affecting hypothalamic function, have paved the way for current and emerging, therapies specifically targeting appetite regulation. These conditions are typically marked by severe early onset obesity, hyperphagia, and a combination of endocrine and neurodevelopmental comorbidities. Challenges in Management Management of genetic forms of obesity remains particularly challenging, as behavioral interventions are often insufficient, and bariatric surgery may be less effective or even contraindicated in some cases. Current Therapeutic Approaches Recent pharmacological treatments aim to restore energy balance by targeting disrupted pathways, most notably the leptin–melanocortin axis, to enhance satiety and reduce hyperphagia. These targeted therapies mark a significant shift from conventional weight-loss approaches by directly addressing the underlying biological mechanisms driving obesity in affected individuals. Emerging Therapies This review provides an overview of the current therapeutic landscape for genetic and syndromic obesities, including approved treatments such as setmelanotide and semaglutide, as well as emerging investigational drugs. Future Directions and Considerations In addition to outlining their place within evolving treatment algorithms, we discuss future directions, ongoing clinical challenges, and key points of vigilance—including long-term efficacy, safety, access, and the need for integrated, multidisciplinary care within a precision medicine framework. Clearly defining their place in treatment algorithms is essential to integrate these therapies into a precision medicine framework for the management of genetic and syndromic obesities.

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