Rhythm Pharmaceuticals: FDA Approves IMCIVREE for Children 2+

BOSTON, Dec. 20, 2024 – Rhythm Pharmaceuticals, Inc., a biopharmaceutical company specializing in treatments for rare neuroendocrine disorders, announced the U.S. FDA's approval of an expanded use for IMCIVREE® (setmelanotide). This medication can now be administered to children as young as two years old. IMCIVREE is designed to help reduce and maintain weight in individuals with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or specific genetically confirmed deficiencies such as pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiencies.

Bardet-Biedl syndrome and POMC, PCSK1, and LEPR deficiencies are rare conditions tied to the melanocortin-4 receptor (MC4R) pathway. These disorders are characterized by severe appetite issues, known as hyperphagia, and early-onset obesity which often begins before the age of five. IMCIVREE is the first approved medication that targets these specific impairments in the hypothalamic MC4R pathway in both children and adults in the U.S. and Europe.

Dr. Ilene Fennoy, a pediatric endocrinologist and obesity specialist, emphasized the distinct nature of these rare MC4R pathway diseases compared to general obesity. She noted that these conditions are rooted in a pathological hunger due to brain pathway disruptions, leading to early-onset obesity. Dr. Fennoy highlighted the importance of early intervention in childhood obesity to prevent severe long-term health issues, stating that the expanded availability of IMCIVREE offers a crucial targeted treatment for young patients.

Clinical trials have shown that setmelanotide significantly reduces weight and hunger levels. The Phase 3 VENTURE trial's results, published in The Lancet Diabetes & Endocrinology in November 2024, support its efficacy. Common side effects include skin hyperpigmentation, injection site reactions, diarrhea, nausea, and headaches.

Tim Ogden, President of the Bardet Biedl Syndrome Foundation, welcomed the FDA's approval, noting its significance for those struggling with hyperphagia. He explained the immense pressure on families dealing with children who constantly feel hungry or think about food, often leading to behaviors like sneaking or stealing food. Ogden emphasized that IMCIVREE provides a vital tool for managing obesity in these patients.

David Meeker, M.D., Chairman and CEO of Rhythm Pharmaceuticals, reiterated the company's dedication to ensuring those affected by rare genetic diseases have timely access to IMCIVREE. He stressed Rhythm's commitment to advancing care and developing precise treatments targeting the root causes of rare neuroendocrine diseases.

Initially approved by the FDA in November 2020 for patients aged six and older with POMC, PCSK1, or LEPR deficiencies, IMCIVREE received authorization in June 2022 for BBS patients. It has also been approved by the UK’s Medicines & Healthcare products Regulatory Agency (MHRA) and the European Commission (EC) for use in children as young as two.

Bardet-Biedl syndrome affects approximately 4,000 to 5,000 individuals in the U.S., manifesting in symptoms such as hyperphagia and severe obesity from an early age, alongside potential cognitive and physical impairments. POMC, PCSK1, and LEPR deficiency obesities, caused by specific genetic variants, impact about 600 to 2,500 people in the U.S.

Rhythm Pharmaceuticals, based in Boston, remains committed to developing therapies for rare neuroendocrine diseases. Their leading product, IMCIVREE, aims to address hyperphagia and severe obesity. The company is also exploring additional treatments for other rare conditions, advancing clinical programs for setmelanotide, and investigating new MC4R agonists and preclinical small molecules for congenital hyperinsulinism.