An Experimental Treatment for POMC and LEPR Deficiency Obesity Appears Effective

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company’s experimental drug setmelanotide as a treatment for two different genetic disorders: LEPR deficiency obesity and POMC deficiency obesity. In both studies, the drug was able to achieve all primary and secondary endpoints, indicating that it could be a very useful treatment for these genetically-linked forms of obesity.

About LEPR Deficiency Obesity

Leptin receptor (LEPR) deficiency obesity is characterized by obesity that becomes noticeable within the first several months of life. Most patients are born with normal weight, but they experience extreme hunger that causes them to gain weight quickly. The disorder is caused by mutations affecting the LEPR gene. The gene is responsible for the production of the leptin receptor protein, which plays an essential role in regulating body weight. To learn more information about LEPR deficiency obesity, click here.

About POMC Deficiency Obesity

Proopiomelanocortin (POMC) deficiency obesity is a rare genetic disorder which causes obesity at a very early age. Only about 50 cases have been reported so far, so it is incredibly rare. It is also characterized by reduced levels of adrenocorticotropic hormone (ACTH). Many people with the disorder have pale skin and red hair. As the name of the illness suggests, the disorder is linked to mutations of the POMC gene. To learn more information about POMC deficiency obesity, click here.

Trial Results

During this trial 8 out of 10 POMC deficiency obesity patients were able to reach the primary endpoint using setmelanotide; the endpoint was more than ten percent weight reduction over the course of a year. Mean weight loss for these patients during the course of the trial was 70.2 lb, with mean weight reduction percentage of 25.4 percent. Patients also saw improvements in self reported hunger scores (50 percent saw 25 percent or greater improvement) and hunger rating (reduced by a mean of 27.8 percent).

Meanwhile 5 of 11 patients with LEPR deficiency obesity met the primary endpoint, which was the same in both studies. Mean weight reduction percentage for this group was 12.5 percent. 72.7 percent saw 25 percent or better improvement in self reported hunger scores and hunger rating dropped by a mean of 41.9 percent. Mean weight loss for LEPR deficiency obesity patients was 36.8 lb. 

The vast majority of patients opted to continue treatment in an open label extension study. With these encouraging results, setmelanotide may very well be on its way to approval.


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