Rhythm Pharma nabs rare ped disease tag for obesity med

The FDA has granted Rare Pediatric Disease Designation to Rhythm Pharmaceuticals’ (NASDAQ:RYTM) setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.

POMC is an inherited disorder characterized by severe obesity due to overeating to satisfy chronic hunger.

LEPR is also characterized by severe obesity due to same urge to satisfy chronic hunger.

Rare Pediatric Disease Designation provides for the issuance of a rare pediatric disease priority review voucher following FDA approval. The voucher can be used for accelerated approval of a future application or it can be sold to a third party.

Setmelanotide activates a protein called melanocortin 4 receptor (MC4R) that plays a key role in controlling appetite.

https://seekingalpha.com/news/3587685-rhythm-pharma-nabs-rare-pediatric-disease-tag-for-setmelanotide-for-obesity-disorders