Setmelanotide Approved: Is This the Future?

This is the ultimate Friday news dump. FDA approved a highly innovative, highly targeted treatment for obesity – setmelanotide – Wednesday night. Just before a four-day holiday. The company announced in on Friday – timing one would typically choose for news they want to hide. But this is really good news for the treatment of obesity. In fact, it might be that this approval gives us a glimpse of the future of obesity therapy.

This drug has a daunting brand name – Imcivree. We’ll have to work on pronouncing it without stumbling. It is a subcutaneous, daily injection – like insulin or Saxenda.

Highly Targeted for Children and Adults

This approval is an important milestone in two ways. First of all it is highly targeted for persons with obesity due to specific genetic defects. These include POMC, PCSK1, and leptin receptor deficiencies. This makes an important point very real. One size does not fit all in obesity. Obesity has many different causes and different people respond very differently to a given therapy because of that.

Folks who think in outmoded ways about obesity have a tough time wrapping their brains around this fact. But as more targeted and effective approaches come along, perhaps reality will displace such simplistic thinking.

Because this therapy is so targeted it is can be more effective than many other drugs. The FDA-approved prescribing information tells us that patients with POMC deficiency lost an average of 27 percent of their starting body weight. Eight out of ten patients lost more than ten percent.

In leptin receptor deficiency, the results were more modest – an average of ten percent weight loss.

Indicated for Children Six and Older

The second reason this is such an important milestone is that the approval is for use in children as young as six years. No other drug has ever been approved for any form of obesity in children. The closest thing is Orlistat, which is for youth down to the age of 12. Its efficacy is modest and its use is not common.

Having a highly effective drug for kids with very specific and severe forms of obesity will open some eyes. This makes the point that obesity is not merely a behavioral problem. It has a biological basis. Kids who struggle with severe obesity now have another option. Setmelanotide might or might not be right for them, but it’s an option to consider. Until now, they had nothing but eat less, move more, and surgery in some cases.

Keeping It Low Key

The interesting aspect of this approval is how quiet it has been. It makes sense if you consider that setmelanotide is a fairly esoteric therapy for rare genetic forms of obesity. However, our view is that rare diseases matter. A breakthrough in targeted therapy is indeed important.

This will be part of a much larger developing story about obesity care for kids and more targeted approaches to obesity therapy generally. We recommend you pay attention.

Click here and here for press releases from Rhythm Pharmaceuticals and the FDA. For the full prescribing information, click here. Finally, you can find further perspective here and here.

Neuron Development, photograph by K. Francis from the NIH Image Gallery / flickr

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November 28, 2020