Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients

Rhythm Pharmaceuticals (Nasdaq: RYTM) announced public reimbursement agreements for IMCIVREE (setmelanotide) in Ontario, Alberta, British Columbia, Saskatchewan, Nova Scotia and with the Federal Non-Insured Health Benefits (NIHB) program for weight management in eligible patients aged 6 years and older with clinically or genetically confirmed Bardet-Biedl syndrome (BBS) and obesity. IMCIVREE was approved by Health Canada in May 2023 after priority review and was recently added to the Common Drug List for rare diseases under Canada’s National Strategy for Drugs for Rare Diseases, supporting broader public access.

Setmelanotide TFA (RM-493 TFA) is a selective MC4R agonist that demonstrates high potency in both human and rat receptor assays. It is a valuable research tool for investigating metabolic regulation, obesity pathways, and energy homeostasis.

Rhythm Pharmaceuticals (NASDAQ:RYTM) announced significant regulatory progress for setmelanotide in treating acquired hypothalamic obesity. The FDA has accepted the company's sNDA with Priority Review, setting a PDUFA date of December 20, 2025. Additionally, the EMA validated the Type II variation submission for the same indication.The applications are supported by the Phase 3 TRANSCEND trial results, which demonstrated a significant -19.8% placebo-adjusted reduction in BMI. The trial showed strong efficacy in both adult patients (-19.2% BMI reduction) and pediatric patients (-20.2% BMI reduction). Setmelanotide, already approved as IMCIVREE® for other rare genetic obesity conditions, was generally well-tolerated in the study.

Rhythm Pharmaceuticals ((RYTM)) announced an update on their ongoing clinical study.

Setmelanotide (CAS 920014-72-8) is a highly selective melanocortin-4 receptor (MC4R) agonist peptide with an EC₅₀ around 0.27 nM. Manufactured under GMP conditions with purity ≥99.93%, it is ideal for research in monogenic obesity, appetite regulation, and energy metabolism. Laboratory research use only.（For wholesale prices, other specifications and uses, please consult our staff）

Setmelanotide was associated with significant weight reduction among patients aged 4 years and older with acquired hypothalamic obesity.

Rhythm Pharmaceuticals (NASDAQ:RYTM) presented significant clinical data for two MC4R agonists - setmelanotide and bivamelagon - in treating acquired hypothalamic obesity at ENDO 2025. The Phase 2 SIGNAL trial for bivamelagon showed significant BMI reductions of up to 9.3% in the highest dose cohort. The Phase 3 TRANSCEND trial for setmelanotide demonstrated a remarkable 19.8% placebo-adjusted BMI reduction across 120 patients.Notably, setmelanotide showed consistent efficacy across age groups and genders, with significant results even in patients using GLP-1s. Patients with prior and concomitant GLP-1 use achieved a 25.1% BMI reduction compared to 2.0% in the placebo group. In patients under 18, the treatment led to a 26.2% reduction in the 95th percentile BMI at week 52.

-- Bivamelagon achieved BMI reductions in patients with acquired hypothalamic obesity of -9.3% and -7.7% in 600mg and 400mg cohorts, respectively, at 14 weeks -- -- Post-hoc analysis showed BMI reduct...

Rhythm Pharmaceuticals' Imcivree shows promise in treating obesity from brain damage, reducing BMI by 16.5% in Phase 3 trial; regulatory filings planned for 2025.

Key efficacy data from TRANSCEND trial evaluating setmelanotide in hypothalamic obesity to be revealed. Breakthrough potential for rare disease treatment.