Rhythm Pharmaceuticals IMCIVREE® for Obesity and Hunger Control Receives NICE Backing

Global biopharmaceutical company, Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), has received a positive recommendation from the National Institute for Health and Care Excellence (NICE) , which has issued guidance for the company’s IMCIVREE® (setmelanotide) as an option for treating obesity and the control of hunger in Bardet-Biedl syndrome (BBS) patients aged 6 years and over. Results from Rhythm’s clinical trials suggest that setmelanotide may reduce hyperphagia, weight and body mass index (BMI).

IMCIVREE is expected to be funded and available for use in England and Wales within three months through NICE in specialist centers.

Commenting on the news, Yann Mazabraud, Executive Vice President, Head of International at Rhythm Pharmaceuticals, said, “Access to IMCIVREE in England and Wales is an important milestone in our global efforts to bring this medicine to patients and families with BBS. We are pleased with this positive recommendation from NICE, made possible by the collaboration and support of advocacy leaders at BBS U.K. and clinical experts, and engaging and productive discussions with NICE.”

BBS is a rare genetic disease characterized by insatiable hunger and severe obesity beginning early in life.

“BBS is a debilitating rare disease that severely affects quality of life of patients and their families,” said Philip Beales, M.D., UCL Great Ormond Street Institute of Child Health. “Hyperphagia – the feeling of extreme hunger that stays with patients all the time – leads to early-onset, life-long, severe obesity that affects many aspects of daily living. Until now there have been no licensed treatments for obesity and hyperphagia caused by BBS.”

According to the NICE recommendation, patients may remain on reimbursed setmelanotide as adults while they continue to benefit from therapy.

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.