Rhythm Pharmaceuticals announced on March 19, 2026, that the FDA approved an expanded indication for IMCIVREE (setmelanotide) to reduce excess body weight and maintain reduction long-term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity. This marks the first FDA-approved therapy for this rare disease, supported by Phase 3 TRANSCEND trial results showing -18.4% placebo-adjusted BMI reduction.
Key takeaways
FDA approved expanded indication for IMCIVREE (setmelanotide) on March 19, 2026, for acquired hypothalamic obesity (HO) in adults and pediatric patients aged 4 years and older.
Approval based on Phase 3 TRANSCEND trial (N=142) with -18.4% placebo-adjusted BMI reduction at 52 weeks (p<0.0001); setmelanotide arm -15.8% (n=94) vs. placebo +2.6% (n=48).
First and only FDA-approved therapy for acquired HO, estimated 10,000 patients in U.S.
Most common adverse reactions (≥20%): skin hyperpigmentation, nausea, vomiting, headache.
IMCIVREE now also indicated for BBS, POMC/PCSK1/LEPR deficiency (prior approvals).
Conference call held March 19, 2026, at 5:00 p.m. ET; presentation available at ir.rhythmtx.com.