CHMP Backs Rhythm Pharma’s IMCIVREE for Young Patients with Obesity Due to Genetic Conditions

Rhythm Pharmaceuticals, Inc., a biopharmaceutical company listed on Nasdaq (RYTM), has made a significant announcement regarding its leading product, IMCIVREE® (setmelanotide). The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the expansion of marketing authorization for IMCIVREE to include children between the ages of 2 and 6 who suffer from obesity due to Bardet-Biedl syndrome (BBS) or deficiencies in pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR).

Yann Mazabraud, Executive Vice President and Head of International at Rhythm Pharmaceuticals, emphasized the company’s ongoing collaboration with health authorities across the European Union. The goal is to ensure that IMCIVREE is accessible to patients struggling with uncontrolled hunger and severe early-onset obesity tied to these rare neuroendocrine disorders. Mazabraud highlighted that the positive CHMP opinion for pediatric use underscores the pressing need to address disruptions in the MC4R pathway and the significant unmet medical needs of young children affected by these conditions. Early diagnosis is crucial to prevent the onset of severe obesity-related comorbidities.

IMCIVREE is the first treatment option authorized in the European Union specifically for controlling hunger and treating obesity in adults and children aged 6 and older with BBS or deficiencies in POMC, PCSK1, or LEPR. The CHMP’s recent opinion recommending the inclusion of children as young as 2 years old will now be reviewed by the European Commission (EC), which has the authority to grant marketing authorizations within the EU. A final decision regarding the expanded marketing authorization for setmelanotide is expected in the latter half of 2024.

Rhythm Pharmaceuticals is dedicated to transforming the lives of patients and their families living with rare neuroendocrine diseases. The company's flagship asset, IMCIVREE (setmelanotide), is an MC4R agonist designed to treat hyperphagia and severe obesity. It is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in both adult and pediatric patients aged 6 and older with monogenic or syndromic obesity due to POMC, PCSK1, or LEPR deficiency, as confirmed by genetic testing. Additionally, it is approved for patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS).

In addition to FDA approval, both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for treating obesity and controlling hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1 deficiency, or biallelic LEPR deficiency in adults and children aged 6 and older.

Rhythm Pharmaceuticals is also advancing a comprehensive clinical development program for setmelanotide targeting other rare diseases. This includes investigational MC4R agonists such as LB54640 and RM-718, along with a preclinical suite of small molecules aimed at treating congenital hyperinsulinism. The company is headquartered in Boston, MA.