Setmelanotide  Hydrochloride

-- Launch follows MHLW approval of IMCIVREE for acquired hypothalamic obesity and inclusion on Japan's National Health Insurance drug price list -- -- IMCIVREE is the first approved therapy in Japan for patients living with acquired hypothalamic obesity -- Oct. 13, 2026 -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced the commercial launch of IMCIVREE® (setmelanotide) in Japan to treat patients living with acquired hypothalamic obesity (HO), following its inclusion on Japan's National Health Insurance (NHI) drug price list. IMCIVREE is the first approved therapy in Japan for acquired HO. "This launch marks an important advancement for people living with acquired HO in Japan where there were no approved therapies for this disease until now," said Yann Mazabraud, Executive Vice President and Head of International at Rhythm. “We have been building our team in Japan in preparation for this launch and are excited about the opportunity to positively impact the lives of Japanese patients. This is a significant milestone in our global efforts to bring innovative therapies to patients with rare neuroendocrine diseases around the world." The launch follows approval by Japan's Ministry of Health, Labour and Welfare (MHLW) of IMCIVREE for acquired hypothalamic obesity in August of this year. The approval was supported by data from the pivotal Phase 3 TRANSCEND trial, which enrolled 142 patients with acquired HO, including 12 patients from a Japanese cohort and 10 supplemental patients enrolled in addition to the primary 120-patient pivotal cohort. The global study met its primary endpoint, demonstrating a statistically significant 18.8% placebo-adjusted reduction in body mass index (BMI). As previously announced, setmelanotide received orphan drug designation from the MHLW for the treatment of acquired hypothalamic obesity. Rhythm estimates that approximately 5,000 to 8,000 people are living with acquired HO in Japan. Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and control of hunger in patients 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment; and for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above, and for the treatment of obesity and control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician experienced in the diagnosis and management of rare forms of obesity with genetic or hypothalamic origin. Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity. The content above comes from the network. if any infringement, please contact us to modify.