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Innovation Observatory > Reports > Drugs > Setmelanotide for obesity in Bardet-Biedl syndrome and Alström syndrome

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Setmelanotide for obesity in Bardet-Biedl syndrome and Alström syndrome


Endocrine, Nutritional and Metabolic

February 2021

Setmelanotide is in clinical development as a first-line treatment for patients with Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) who develop an increased appetite (hyperphagia) and are obese. BBS and AS are genetic conditions that impact multiple body systems. Both syndromes are caused by genetic mutations that are usually inherited from their parents. Obesity is a key symptom of both syndromes. The current treatment option is limited to only managing symptoms; no pharmacological options are currently available.
Setmelanotide is a protein that binds to a specific receptor to activate areas in the brain that control appetite. This reduces hunger sensations and therefore obesity. This is important in BBS and AS as obesity and hyperphagia are symptoms in both syndromes and these conditions can have damaging effects on the health of patients. If licensed, setmelanotide, given as an injection under the skin, will offer a first pharmacological treatment for BBS and AS.

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