RHYTHM PHARMACEUTICALS, INC

Obesity, linked to 5 million deaths globally, increases risks of T2DM, heart disease, and cancers. GLP1R agonists like semaglutide (WEGOVY) emerged as effective treatments, with tirzepatide (ZEPBOUND) and setmelanotide (IMCIVREE) also approved. The article updates on current obesity treatments and ongoing clinical trials.

Various drugs and their statuses are listed, including IN-001 for anaphylaxis (Fast Track), Finereonone for heart failure (Phase 3), Tirzepatide for heart failure and obesity (Phase 3), TMB-001 for congenital ichthyosis (Phase 3), Bentracimab for ticagrelor reversal (Priority Review), Fabhalta for immunoglobulin A nephropathy (accelerated approval), RLS-0071 for acute graft-vs-host disease (Fast Track and Orphan Drug), CHIKV VLP for chikungunya (Priority Review), Diazoxide choline for Prader-Willi syndrome (Priority Review), Mirdametinib for neurofibromatosis type 1 (Priority Review), Setmelanotide for genetic obesity (Priority Review), AR882 for gout (Fast Track), SPN-830 for Parkinson disease (NDA review), BGB-16673 for chronic lymphocytic leukemia (Fast Track), Cabozantinib for neuroendocrine tumors (NDA review), Linvoseltamab for multiple myeloma (Complete Response Letter), Nivolumab plus ipilimumab for hepatocellular carcinoma (BLA review), Midomafetamine for PTSD (Complete Response Letter), and Tezepelumab-ekko for COPD (Breakthrough Therapy).

Rhythm Pharmaceuticals seeks FDA approval to expand IMCIVREE® (setmelanotide) use for treating obesity in children as young as 2 with Bardet-Biedl syndrome or POMC/LEPR deficiency, with a PDUFA goal date of December 26, 2024. The sNDA is based on Phase 3 trial data showing efficacy in reducing BMI in young children.