Rhythm Pharmaceuticals has received approval from Japan's Ministry of Health, Labour and Welfare for IMCIVREE (setmelanotide) to treat patients with acquired hypothalamic obesity (HO). This marks the first therapy approved in Japan for this condition. The approval was supported by the Phase 3 TRANSCEND trial involving 142 patients, including a Japanese cohort. The study met its primary endpoint with an 18.8% placebo-adjusted reduction in body mass index. Rhythm estimates 5,000 to 8,000 people are living with acquired HO in Japan. The company expects to launch IMCIVREE before the end of 2026, pending final pricing determination. Acquired HO is characterised by accelerated weight gain following hypothalamic injury, often resulting from craniopharyngioma, astrocytoma, or other hypothalamic-pituitary tumours.

Rhythm Pharmaceuticals has appointed Manher "AJ" Joshi as Chief Medical Officer, effective 17 August. Dr Joshi brings over 20 years of experience spanning drug discovery, pivotal trials, regulatory processes and lifecycle management. Dr Joshi joins from Soleno Therapeutics, where he served as Chief Development Officer. He previously held the Chief Medical Officer position at Neurona Therapeutics and executive roles at Atara Biotherapeutics. His career also includes leadership positions at Allergan Medical, Synageva BioPharma, and Genzyme. Rhythm is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases. Its lead asset, IMCIVREE (setmelanotide), is approved in the US and Europe for treating certain forms of obesity. The company is advancing next-generation MC4R agonists through late-stage development.

Rhythm Pharmaceuticals announced that the UK's Medicines and Healthcare products Regulatory Agency has expanded the marketing authorisation for IMCIVREE (setmelanotide) to include treatment of obesity and control of hunger in adults and children aged 4 and older with acquired hypothalamic obesity due to hypothalamic injury or impairment. The MHRA also granted IMCIVREE orphan drug designation for acquired HO. The approval is based on results from the Phase 3 TRANSCEND trial, which met its primary endpoint with a statistically significant 19.8% placebo-adjusted reduction in body mass index. The global trial enrolled 120 patients. IMCIVREE initially received marketing authorisation in the UK in 2021 for treatment of obesity in patients with POMC, PCSK1 or LEPR deficiency, with subsequent expansions for other rare obesity conditions.