This Drug Would Target the Heart of Genetic Obesity

Rhythm Pharmaceuticals is currently developing a melanocrotin-4 (MC4) receptor therapy called setmelanotide for the treatment of obesity and related disorders caused by genetic defects.

This therapy could potentially be used to treat ROHHAD, or rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation, a rare disorder that affects children and currently has no treatment. Although the cause of ROHHAD is still largely unknown, many doctors attribute the disorder to faulty genetics. To learn more about ROHHAD, click here.

After the U.S. FDA approved the expansion of a Breakthrough Therapy designation (BTD) for setmelanotide, Rhythm began a Phase 3 clinical trial of setmelanotide last month.

Setmelanotide works by targeting the MC4 pathway, which is a key biological passage responsible for regulating weight by increasing energy expenditure and inhibiting appetite. Thus, setmelanotide may be able to fix the underlying cause of obesity disorders, including ROHHAD.

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The Phase 3 trial will focus on evaluating the safety and efficacy of setmelanotide in patients who have POMC deficiency obesity, ROHHAD’s rare cousin. These patients will get setmelanotide administered to them once daily for 12 months, so the initial results of the study should be available by mid-2018.

Because there are currently no approved therapies for genetic disorders of obesity, there is a huge urgency in this sector of the rare disease community.

Luckily, plans are in motion to address these conditions and finally find not only a treatment, but a cure.

To learn more about setmelanotide and Rhythm Pharmaceuticals, click here.

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