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Setmelanotide - First Drug to Treat Rare Genetic Causes of Severe Obesity Approved by FDA

by Aishwarya Nair on December 4, 2020 at 8:51 AM
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Setmelanotide - First Drug to Treat Rare Genetic Causes of Severe Obesity Approved by FDA

Setmelanotide has been approved by the US Food and Drug Administration (FDA) for weight management in adults and children as young as 6 years of age. It is used for treating obesity that is confirmed by genetic testing and caused due deficiency of proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) gene, or leptin receptor (LEPR) .

Individuals with these rare genetic causes of severe obesity have a normal weight at birth. It is seen that they develop persistent, severe obesity within a short span of some months due to unsatisfied hunger. This leads to increased tendency of eating that results into overeating.


Setmelanotide is a melanocortin - 4 receptor agonist (activates that receptor and promotes it's biological activity). Setmelanotide is the first FDA approved therapy for such rare genetic disorder that causes obesity.

" Many patients and families who live with these diseases face an often burdensome stigma associated with severe obesity. To manage this obesity and control disruptive food-seeking behavior, caregivers often lock cabinets and refrigerators and significantly limit social activities, " said Jennifer Miller, MD, a pediatric endocrinologist at University of Florida Health, Gainesville, in a press release issued by the company.

She also stated," This FDA approval marks an important turning point, providing a much needed therapy and supporting the use of genetic testing to identify and properly diagnose patients with these rare genetic diseases of obesity ."

David Meeker, MD, chair, president, and chief executive officer of Rhythm Pharmaceuticals, added: " We are advancing a first-in-class, precision medicine that is designed to directly address the underlying cause of obesities driven by genetic deficits in the MC4R pathway. "

Two phase 3 clinical trials were conducted for the evaluation of Setmelanotide. In the first trial it was observed that 80% of patients with obesity due to POMC or PCSK1 deficiency achieved greater than 10% weight loss after 1 year of treatment. The second trial showed 45.5% of patients with obesity due to LEPR deficiency achieved greater than 10% weight loss with 1 year of treatment. The results for these trials have been published in The Lancet Diabetes & Endocrinology.

Setmelanotide was well-tolerated in both the trials. The most common side effects of the drug are injection site reaction, skin hyperpigmentation, nausea, depression, suicidal tendency, darkening of birth mark or mole.

Benzyl alcohol is used as a preservative in this formulation hence should be used with caution in neonates and low birth weight infants. Setmelanotide has been given FDA breakthrough therapy designation as well as orphan drug designation. It is being marketed by by Rhythm Pharmaceuticals as Imcivree.

Source: Medindia

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