This week, Rhythm Pharmaceuticals announced that a new, targeted obesity medication may soon be available based on their latest research.

The data came from two studies of the drug setmelanotide, which was used to treat rare genetic forms of obesity. Rhythm expects to file for FDA-approval in late 2019 or early 2020. This means that this new treatment for rare forms of obesity may be available by the end of 2020.

Rare Diseases, Small Studies on Obesity Treatment

Let’s be clear. Rhythm has very targeted research. They’ve studied two very rare forms of obesity:

  1. Lepton Receptor (LEPR) – Affects no more than 2,000 U.S. patients
  2. POMC Deficiency – Affects no more than 500 U.S. patients

These diseases occur in young children. Therefore, the studies focus on children between six and eleven years old. Yes, the studies are very small. However, the results are impressive.

In POMC Deficiency:

  • 80% of patients lost more than 10% of their starting weight
  • On average, patients lost 25% of their weight

In LEPR Deficiency:

  • 45% of patients saw 10% or more weight-loss
  • On average, patients lost 12.5% of their weight
  • These results were strong, but less impressive

These are rare diseases that are nearly impossible to treat. Because of this, the FDA has named setmelanotide a breakthrough treatment. Open label, uncontrolled studies were allowed. For patients with these conditions, spontaneous recovery is unheard of.

A Risky Road to Important Innovation

Without a doubt, developing a drug for targeted obesity therapy in rare genetic conditions is risky. Zafgen went down a similar path with a new drug and hit a dead end in 2016.

Though new drugs are risky, they are important. Hopefully Rhythm’s new drug will bring success for young patients. This is innovation that some kids with severe obesity really need.

Click HERE and HERE for more information on the study and its results.