NICE Makes Positive Recommendation on Rhythm’s Imcivree

Rare Daily Staff

The U.K.’s National Institute for Health and Care Excellence has issued guidance that recommends Rhythm Pharmaceuticals Imcivree as an option for treating obesity and hyperphagia in genetically confirmed Bardet-Biedl syndrome in people 6 to 17 years of age when treatment starts.

The final NICE recommendation is aligned to the European Marketing Authorization and U.K. Medicines and Healthcare Products Regulatory Agency approval. With this recommendation under the Highly Specialised Technologies pathway, Imcivree is expected to be funded and available for use within three months in the National Health Service covering England and Wales.

Bardet-Biedl syndrome (BBS) is a rare genetic disease with an estimated prevalence of approximately 900 patients in England and Wales. People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. Results from clinical trials suggest that Imcivree may reduce hyperphagia, weight, and body mass index in people aged 6 years and over. The most common adverse events are skin hyperpigmentation, injection site reactions, nausea, and headache.

Imcivree targets the MC4 receptor pathway, a neural pathway responsible for regulating hunger, energy expenditure, and body weight. The genetic variants in people with these conditions impair the MC4 receptor pathway and as a result they feel extreme, insatiable hunger, and that causes early-onset, severe obesity.

“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,” said Yann Mazabraud, executive vice president and head of international at Rhythm Pharmaceuticals. “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.”

Northern Ireland is expected to adopt NICE guidance. And Rhythm said it is moving ahead with submission to the Scottish Medicines Consortium with a decision expected in 2025.

Both the European Commission and the UK’s Medicines and Healthcare Products Regulatory Agency have authorized Imcivree 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 Imcivree 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.

In the United States, Imcivree is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS) or BBS.

In the European Union and United Kingdom, Imcivree 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 6 years of age and above. In the European Union and United Kingdom, Imcivree should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.

“BBS is a debilitating rare disease that severely affects quality of life of patients and their families,” said Philip Beales, 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.”