FDA Accepts Priority Review for IMCIVREE® in Patients as Young as 2

Rhythm Pharmaceuticals, Inc., a global biopharmaceutical firm specializing in rare neuroendocrine diseases, has achieved a significant milestone. The U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide). IMCIVREE®, a melanocortin-4 receptor (MC4R) agonist, is sought for treating obesity in young children aged 2 and above who suffer from Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The FDA has designated this application for Priority Review and has set a Prescription Drug User Fee Act (PDUFA) target date of December 26, 2024.

David Meeker, M.D., Chairman, CEO, and President of Rhythm, emphasized the importance of this development, stating, "This milestone brings us another step closer to offering a precision therapy for young patients in the U.S. experiencing hyperphagia—a pathological hunger that leads to aberrant food-seeking behaviors—and severe obesity caused by rare MC4R pathway diseases.” IMCIVREE® has been on the market for over two years and has been prescribed to numerous patients aged 6 and older. Extending the treatment to even younger children could significantly impact these children's and their families' lives.

The sNDA submission is grounded on data from Rhythm's multi-center, one-year, open-label Phase 3 trial involving 12 patients aged between 2 and 6 years. These patients had obesity due to biallelic POMC/PCSK1 or LEPR deficiency, or a clinical diagnosis of BBS. The data demonstrated potential efficacy in combating hyperphagia and severe obesity in genetically-caused MC4R pathway diseases that manifest early in life. Treatment with setmelanotide in this study resulted in a primary endpoint achievement, showing a 3.04 mean reduction in BMI-Z score and an 18.4 percent mean reduction in BMI.

In addition to the developments in the U.S., in July 2024, the European Commission (EC) extended the marketing authorization for IMCIVREE® to include children as young as 2 years old with obesity due to BBS or POMC/PCSK1, or LEPR deficiency. In the United States, IMCIVREE® is currently approved for chronic weight management in adults and pediatric patients 6 years and older with monogenic or syndromic obesity due to POMC, PCSK1, or LEPR deficiency. This approval requires an FDA-approved test confirming pathogenic or likely pathogenic variants in these genes, as well as for those with a clinical diagnosis of BBS.

Rhythm Pharmaceuticals is dedicated to improving the lives of patients with rare neuroendocrine diseases. The company's primary product, IMCIVREE® (setmelanotide), targets hyperphagia and severe obesity and is approved by the FDA for chronic weight management in patients 6 years and older with specific genetic conditions or BBS. The European Commission and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have also approved setmelanotide for treating obesity and controlling hunger in patients with genetically confirmed BBS or loss-of-function biallelic POMC, PCSK1, or LEPR deficiency in adults and children aged 6 and above. The EC has further authorized the drug for children as young as 2 with these conditions.

Rhythm is also pushing forward with an extensive clinical development program for setmelanotide in other rare diseases. This includes investigational MC4R agonists LB54640 and RM-718, along with a preclinical suite of small molecules aimed at treating congenital hyperinsulinism. Headquartered in Boston, MA, Rhythm remains committed to transforming the treatment landscape for patients with rare neuroendocrine disorders.