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.
Tirzepatide is a new type of drug that is administered by subcutaneous injection and acts on two proteins known as the gastric inhibitory peptide (GIP) and glucagon-like peptide-1 (GLP-1) and results in more insulin being released from the pancreas. Tirzepatide has the potential to improve blood sugar levels and increase weight loss compared to current treatment options. If licenced, tirzepatide will offer an additional treatment option for patients with T2DM.