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EMA grants Orphan Drug Designation for Setmelanotide for treatment of acquired hypothalamic obesity

The European Medicines Agency (EMA) has issued a positive opinion on the Rhythm Pharmaceuticals’ orphan drug designation request for setmelanotide (IMCIVREE) as a treatment for acquired hypothalamic obesity. EMA orphan drug designation is granted to drugs intended for the treatment, diagnosis, or prevention of life-threatening or chronically debilitating conditions affecting no more than five in 10,000 individuals in the European Union.

Rhythm is currently evaluating setmelanotide in a global Phase 3 clinical trial in acquired hypothalamic obesity and expects to complete patient enrolment in the fourth quarter of 2023.


Acquired hypothalamic obesity is a rare form of extreme obesity that occurs following damage to the hypothalamic region of the brain, which includes the melanocortin-4 receptor (MC4R) pathway and is responsible for controlling physiological functions such as hunger and weight regulation. It most frequently follows the growth or surgical removal of craniopharyngioma, astrocytoma, or other rare brain tumours.

Patients experience rapid weight gain, a reduction in energy expenditure, and an increase in hunger leading to severe obesity within six to 12 months following tumour resection. Rhythm estimates there are approximately 3,500 to 10,000 patients living with acquired hypothalamic obesity in the European countries of Germany, France, Spain, Italy, the Netherlands, and the UK.


Setmelanotide is approved in the US and authorized by the European Commission and the UK’s Medicines & Healthcare Products Regulatory Agency for use in accordance with product labelling. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare MC4R pathway diseases, as well as a preclinical suite of investigational candidates for the treatment of congenital hyperinsulinism.


“Acquired hypothalamic obesity is a serious disease with severe implications for patients and families and no effective treatment options,” said Yann Mazabraud, Executive Vice President and Head of International at Rhythm. “We are pleased with this EMA positive opinion as it underscores the significant unmet need for these patients and the potential of setmelanotide to bring a precision medicine approach that may benefit patients with this disease across Europe.”

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