The U.S. Food and Drug Administration (FDA) has approved Rhythm Pharmaceuticals’ setmelanotide for chronic weight management in patients 6 years and older with obesity due to the following three rare conditions: pro-opiomelanocortin (POMC) deficiency, proprotein subtilisin/kexin type 1 (PCSK1) deficiency, and leptin receptor (LEPR) deficiency.
Setmelanotide has been approved for individuals where genetic testing demonstrates variants in POMC, PCSK1, or LEPR genes are pathogenic, likely pathogenic, or of uncertain significance. It is the first FDA-approved treatment for POMC, PCSK1, or LEPR deficiency.
Individuals with obesity due to these conditions become obese at a young age due to a genetic defect that affects their hunger level, ability to feel satiated, and metabolism. POMC, PCSK1, and LEPR deficiency are very rare – only about 150 cases have been reported for all three conditions combined.
Setmelanotide works by activating areas in the brain that regulate appetite and fullness, causing patients with specific defects in these areas of the brain not to eat as much. The drug also increases resting metabolism. However, setmelanotide does not treat the genetic defects that cause POMC, PCSK1, or LEPR deficiency.
The approval of setmelanotide was largely due to the results of two studies. The first study enrolled patients 6 years or older with obesity with confirmed or suspected POMC or PCSK1 deficiency. The results showed that 80% of these patients lost 10% or more of their body weight. The second study enrolled patients 6 years or older with obesity and confirmed or suspected LEPR deficiency. The results of this study found that 46% of these patients lost 10% or more of their body weight. Both studies also assessed maximal hunger and found that some patients had a decrease in their weekly maximal hunger score.
The most common side effects of setmelanotide include injection site reactions, skin hyperpigmentation, headache and gastrointestinal side effects (such as nausea, diarrhea, and abdominal pain), among others.
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