Rhythm Pharmaceuticals Wins FDA Nod for IMCIVREE in Acquired Hypothalamic Obesity
Rhythm Pharmaceuticals received FDA approval for IMCIVREE as the first therapy to treat acquired hypothalamic obesity in patients aged four and older. Approval follows a Phase 3 TRANSCEND trial showing an 18.4% placebo-adjusted BMI reduction at 52 weeks in 142 patients.
1. FDA Approval for Acquired Hypothalamic Obesity
The FDA has approved IMCIVREE for adults and pediatric patients aged four and older with acquired hypothalamic obesity, marking the first and only therapy for a condition characterized by accelerated, sustained weight gain due to hypothalamic injury or dysfunction.
2. Phase 3 TRANSCEND Trial Results
The global Phase 3 TRANSCEND trial enrolled 142 patients and met its primary endpoint with a placebo-adjusted 18.4% reduction in BMI at 52 weeks; participants on setmelanotide experienced a mean 15.8% BMI decrease versus a 2.6% increase in the placebo group (p<0.0001), with skin hyperpigmentation, nausea, vomiting and headache as the most common adverse events.
3. Target Patient Population
Rhythm estimates approximately 10,000 individuals in the U.S. live with acquired hypothalamic obesity, a patient population most frequently arising from tumors or hypothalamic injury, offering a tangible commercial opportunity for setmelanotide beyond existing monogenic obesity indications.
4. Commercial Launch and Support Programs
IMCIVREE will be available immediately in the U.S., and Rhythm will host a conference call on March 19 to discuss the approval; the company’s InTune support program will provide education, insurance navigation and injection assistance to eligible patients.