Rhythm Pharmaceuticals Receives Positive CHMP Opinion for ▼IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Patients between 2 and 6 years old with Bardet Biedl Syndrome or POMC, PCSK1, or LEPR Deficiency

2024-07-01

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上市批准

- European Commission decision anticipated in the second half of 2024 -

June 28, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the marketing authorization for IMCIVREE® (setmelanotide) include children between 2 and younger than 6 years old with obesity due to Bardet Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.

“We continue to work closely with health authorities throughout the European Union to make IMCIVREE available to eligible patients with uncontrolled hunger and early-onset, severe obesity associated with these rare neuroendocrine diseases,” said Yann Mazabraud, Executive Vice President, Head of International at Rhythm Pharmaceuticals. “We believe this positive CHMP opinion for pediatric use underscores the severe impact of the disruption of the MC4R pathway and significant unmet medical need for young children. It is important to diagnose patients with these diseases early in life before the comorbidities of severe obesity take hold.”

IMCIVREE is the first-ever authorized treatment option in the European Union for control of hunger and treatment of obesity and in adults and children 6 years of age and above living with BBS or POMC, PCSK1, or LEPR deficiency.

The CHMP opinion on the marketing authorization to include patients as young as 2 years old now will be reviewed by the European Commission (EC), which has the authority to grant marketing authorizations for medicinal products in the European Union (EU). A final decision on the application to expand the marketing authorization for setmelanotide is anticipated in the second half of 2024.

About Rhythm Pharmaceuticals

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) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide 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 6 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 LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA.

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