- Commercial-stage biotech Rhythm Pharmaceuticals ( NASDAQ: RYTM ) added ~5% pre-market Tuesday after announcing that the FDA issued Breakthrough Therapy Designation for its lead candidate setmelanotide as a treatment for hypothalamic obesity.
- Setmelanotide, marketed as Imcivree, is already indicated in the U.S. for weight management in conditions including the rare genetic disorder Bardet-Biedl syndrome.
- Hypothalamic obesity is a rare disorder characterized by extreme obesity resulting from a damaged hypothalamus in the brain.
- With its Breakthrough Therapy designation, the FDA aims to speed up the development and review of treatments targeted at serious or life-threatening conditions.
- The designation allows a company to receive intensive regulatory guidance from the FDA during drug development with potential eligibility for priority review.
- The FDA decision is based on data from a Phase 2 16-week clinical trial for the candidate.
For further details see:
Rhythm Pharma gains as FDA issues breakthrough status for weight loss agent