Product Description
Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist in late-stage clinical development for the treatment of rare genetic diseases of obesity by directly addressing the underlying cause of obesities driven by genetic deficits in the MC4R pathway. (Sourced from: https://www.rhythmtx.com/our-pipeline/)
Mechanisms of Action: MCR Agonist
Novel Mechanism: No
Modality: Peptide/Protein
Route of Administration: Subcutaneous
FDA Designation: Breakthrough Therapy - ObesityOrphan Drug - Alstrom SyndromeOrphan Drug - ObesityPriority Review - *
Approval Status: Approved
Approved Countries: Austria | Belgium | Croatia | Czech | Estonia | European Medicines Agency | Finland | France | Germany | Hungary | Iceland | Ireland | Israel | Italy | Latvia | Lithuania | Netherlands | Poland | Portugal | Slovakia | Spain | Sweden | United Kingdom | United States
Approved Indications: Protein C Deficiency | Obesity | Pediatric Obesity | Protein Deficiency
Known Adverse Events: Depressive Disorder | Abdominal Pain | Back Pain | Headache | Pain Unspecified | Hyperpigmentation | Respiratory Tract Infections | Diarrhea
Company: Rhythm
Company Location: BOSTON MA 02116
Company CEO: David P. Meeker
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Canada, France, Germany, Greece, Israel, Netherlands, Puerto Rico, Spain, United Kingdom, United States
Active Clinical Trial Count: 15
Highest Development Phases
Phase 3: Bardet-Biedl Syndrome|Body Weight Changes|Deficiency Diseases|Laurence-Moon Syndrome|Obesity
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| RM-493-042 | P3 |
Enrolling by invitation |
Obesity |
2027-10-30 |
|
| RM-493-035 | P3 |
Unknown Status |
Obesity|Body Weight Changes |
2026-12-31 |
|
| EMANATE | P3 |
Recruiting |
Obesity |
2025-12-01 |
|
| Setmelanotide in patients with specific gene defects | P2 |
Completed |
Unknown |
2025-05-14 |