"This EC authorization is an important step toward making the first new treatment option available to people living with acquired HO in Europe," said David Meeker, M.D., Chairman, President and Chief Executive Officer of Rhythm Pharmaceuticals. "The hyperphagia and accelerated, sustained weight gain due to hypothalamic dysfunction resulting from direct injury or structural abnormality of the hypothalamus has a significant impact patients and families, who until now have had no approved treatment options. With this authorization in place, we look forward to working with local health authorities to support access for patients who may benefit throughout Europe."

Acquired HO is a rare neuroendocrine disease characterized by accelerated and sustained weight gain caused by hypothalamic injury or impairment. Rhythm estimates there are approximately 10,000 people living with acquired HO in Europe.

The EC approval is based on results from the pivotal, 120-patient cohort of the Phase 3 TRANSCEND trial of setmelanotide in acquired HO. The global study met its primary endpoint, with a statistically significant -19.8% placebo-adjusted reduction in body mass index (BMI). For the primary endpoint of mean BMI change from baseline, study participants on setmelanotide therapy (n=81) achieved a -16.5% reduction compared with a +3.3% increase among patients on placebo (n=39) at 52 weeks (p<0.0001). Adult patients (age 18 and older; n=49) achieved a -19.2% placebo-adjusted BMI reduction at 52 weeks, and pediatric patients (younger than age 18; n=71) achieved a -20.2% placebo-adjusted BMI reduction at 52 weeks. Setmelanotide was generally well tolerated in the TRANSCEND study. The most common treatment-emergent adverse events (affecting >20% of participants) were nausea, vomiting, diarrhea, injection site reaction, skin hyperpigmentation and headache.

Please refer to the full Summary of Product Characteristics for a complete list of indications, contraindications, warnings and precautions.

With EU authorization in place, The Company will now work to secure access on a country-by-country basis across Europe. Commercial launches are anticipated in 2027.