LB54640

Setmelanotide Receives Orphan Drug Designation in Japan for Hypothalamic Obesity Treatment

2 months ago

• Rhythm Pharmaceuticals' setmelanotide has received orphan drug designation from Japan's Ministry of Health for the treatment of acquired hypothalamic obesity, a rare condition affecting an estimated 5,000-8,000 patients in Japan. • Setmelanotide, a melanocortin-4 receptor agonist, is currently being evaluated in a global Phase 3 trial with topline data expected in Q2 2025, potentially offering the first therapy targeting the underlying biology of this disease. • The designation follows similar status in Europe, strengthening Rhythm's global strategy to address significant unmet medical needs for patients with hypothalamic obesity caused by brain tumors, traumatic injury, stroke, or inflammation.

FDA Grants Priority Review for Setmelanotide sNDA in Young Children with Rare Genetic Obesity

9 months ago

• The FDA has accepted Rhythm Pharmaceuticals' sNDA for setmelanotide, seeking approval for children aged 2-6 with obesity due to Bardet-Biedl syndrome or POMC/LEPR deficiency. • The sNDA is supported by Phase 3 trial data showing a 3.04 mean reduction in BMI-Z score and an 18.4% mean reduction in BMI with setmelanotide treatment. • The FDA has granted Priority Review with a PDUFA goal date of December 26, 2024, potentially offering a precision therapy for young patients with rare MC4R pathway diseases. • Setmelanotide is currently approved for patients 6 years and older with specific genetic obesity conditions, and this expansion could positively affect younger children and their families.

Setmelanotide Receives Orphan Drug Designation in Japan for Hypothalamic Obesity Treatment

FDA Grants Priority Review for Setmelanotide sNDA in Young Children with Rare Genetic Obesity

Drug Development Updates