FDA gets into Rhythm with a 'breakthrough' title for orphan obesity drug

In the R&D game, obesity and diabetes are two fields that require big studies and a steady if not slow-motion approach to determining a new drug's safety and efficacy. And up until today, the FDA's Division of Metabolism and Endocrinology Products has not singled out any one product for the agency's breakthrough therapy designation, which comes with a commitment to help accelerate development. Little Boston-based Rhythm, though, just broke the mold with a midstage drug aimed at small, genetically defined patient populations.

The FDA has handed Rhythm an unusual BTD for setmelanotide, recognizing that the Phase II drug is aimed at pro-opiomelanocortin (POMC) deficiency obesity, a rare genetic condition that is targeted by the melanocortin-4 receptor (MC4R) agonist. The company is also enrolling a Phase II study of the drug for Prader-Willi syndrome, a rare overeating disorder.

Just last summer Pfizer and Orbimed joined a large syndicate backing a $40 million round for the company. Rhythm has been keeping a low profile since it was founded in 2010 by Bart Henderson and Liz Stoner. Merck ($MRK) R&D vet Keith Gottesdiener was recruited to take the helm in 2011, and the company had raised a total of $73 million by the end of 2012.

"Patients with POMC deficiency obesity have extreme and unrelenting appetite and obesity because of impaired function in the MC4 pathway," said Gottesdiener, CEO of Rhythm, in a statement. "We are pleased to receive this breakthrough designation and look forward to working closely with the FDA as we continue to advance the setmelanotide program."

- here's the release

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