MedPath

Setmelanotide

Generic Name
Setmelanotide
Brand Names
Imcivree
Drug Type
Small Molecule
Chemical Formula
C49H68N18O9S2
CAS Number
920014-72-8
Unique Ingredient Identifier
N7T15V1FUY
Background

Setmelanotide is the first available treatment for patients with pro-opiomelanocortin, proprotein subilisin/kexin type 1, or leptin deficiencies. It is an agonist of the melanocortin 4 receptor. Earlier attempts at agonizing MC4R (such as LY2112688) lead to successful weight loss, but also an increase in blood pressure and heart rate. Other earlier treatments for these patients included gastric bypass surgery. Patients taking setmelanotide experienced an average weight loss of 0.6 kg/week.

Imcivree was granted EMA orphan designation on November 19, 2018 and FDA approval on November 25, 2020. On May 4, 2023, it was approved by Health Canada.

Indication

Setmelanotide is indicated for chronic weight management in patients 6 years and older with obesity due to pro-opiomelanocortin (POMC) deficiency, proprotein subtilisin/kexin type 1 (PCSK1) deficiency, or leptin receptor (LEPR) deficiency as determined by an approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance. These conditions affect the MC4R signalling pathway.

Setmelanotide is also indicated for chronic weight management in patients 6 years and older with obesity due to Bardet-Biedl syndrome.

The drug is not reported to be effective in patients with POMC, PCSK1, or LEPR variants classified as benign or likely benign, as well as other types of obesity not listed above.

Associated Conditions
Leptin Receptor Deficiency Obesity, Obesity, Pro-opiomelanocortin (POMC) Deficiency Obesity, Proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency obesity
Associated Therapies
Chronic Weight Management therapy

Rhythm announces FDA approval of Imcivree for patients as young as 2

Rhythm Pharmaceuticals' IMCIVREE received FDA approval for treating children as young as 2 with rare obesity disorders, targeting the MC4R pathway to address hyperphagia and early-onset obesity, marking a significant advancement in precision medicine for these conditions.
finance.yahoo.com
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Rhythm Pharmaceuticals' Obesity Treatments Target Less Crowded Space, Analyst Sees Long-Term Potential

Oppenheimer initiated coverage on Rhythm Pharmaceuticals Inc. (RYTM), highlighting its focus on rare obesity disorders with Imcivree, showing a 14% revenue increase to $33.3M in Q3 2024. With a $76 price target, optimism is high for its MC4 biology expertise and Phase 3 trial results for hypothalamic obesity expected in 2025.
stockhouse.com
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Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients as Young as 2 Years Old

Rhythm Pharmaceuticals announced FDA approval for IMCIVREE® (setmelanotide) to treat children as young as 2 with rare obesity disorders like BBS and POMC, PCSK1, or LEPR deficiencies. IMCIVREE targets the MC4R pathway, addressing hyperphagia and obesity. Clinical trials show significant weight and hunger reduction. Common side effects include skin hyperpigmentation and injection site reactions.
globenewswire.com
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Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE®

Rhythm Pharmaceuticals announces FDA approval of expanded indication for IMCIVREE® (setmelanotide) to include children as young as 2 years old for syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. IMCIVREE targets the hypothalamic MC4R pathway, reducing excess body weight and maintaining weight reduction long-term.
biospace.com
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FDA Action Alert: Ionis, Lexicon, AstraZeneca/Daiichi Sankyo and More

The FDA is set to decide on ten drug applications, including Ionis' olezarsen for familial chylomicronemia syndrome, Lexicon's sotagliflozin for type 1 diabetes, AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC, Zealand Pharma's glepaglutide for short bowel syndrome, Rhythm Pharmaceuticals' Imcivree for genetic obesity in children, Checkpoint Therapeutics' cosibelimab for cutaneous squamous cell carcinoma, Mirum's chenodiol for cerebrotendinous xanthomatosis, Bristol Myers Squibb's subcutaneous Opdivo, and Neurocrine's crinecerfont for congenital adrenal hyperplasia.
rttnews.com
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Biotech Stocks Facing FDA Decision In December 2024

FDA decisions in December include BeiGene's Tevimbra for gastric cancer, Ionis Pharma's Olezarsen for familial chylomicronemia syndrome, Lexicon Pharma's Zynquista for type 1 diabetes, AstraZeneca's Datopotamab deruxtecan for lung cancer, Zealand Pharma's Glepaglutide for short bowel syndrome, Rhythm Pharma's IMCIVREE for obesity in children, Soleno's DCCR for Prader-Willi syndrome, Checkpoint's Cosibelimab for skin cancer, Mirum's Chenodiol for cerebrotendinous xanthomatosis, Bristol-Myers' subcutaneous Opdivo, and Neurocrine's Crinecerfont for congenital adrenal hyperplasia.
morningstar.com
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Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Study of setmelanotide in Children with Bardet Biedl Syndrome or POMC, PCSK1, or LEPR Deficiency

Rhythm Pharmaceuticals published Phase 3 VENTURE trial results of setmelanotide in children aged 2-5 with Bardet Biedl syndrome or POMC, PCSK1, or LEPR deficiency, showing clinically meaningful reductions in hunger and body weight, published in The Lancet Diabetes & Endocrinology.
investing.com
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HR Chief Pamela Cramer Sells $585,940 in Rhythm Pharmaceuticals Stock

Pamela J. Cramer sold 9,375 Rhythm Pharmaceuticals shares for $585,940, exercised options for 9,375 shares at $19.02 each, and holds 13,500 shares. Rhythm reported a 48% Q3 revenue increase to $33.3M, reduced 2024 operating expense guidance, and has $298.4M in cash reserves. The company expects top-line Phase III trial data in H1 2025 and is optimistic about completing trial enrollments by year-end. RYTM stock has shown 133.08% total return over the past year, but faces financial challenges with an operating income margin of -235.95%.
investing.com
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Earnings call: Rhythm Pharmaceuticals has reported a successful third quarter in 2024

Rhythm Pharmaceuticals reports Q3 2024 success with $33.3 million in global net revenue from IMCIVREE, a 48% YoY increase. The company anticipates funding operations into 2026, with plans to complete Phase II trials for bivamelagon and Phase I for RM-718 in early 2025. Rhythm expects to expand IMCIVREE's label to include children ages two to six by December 26, 2024, and forecasts European revenue growth in 2025.
medicaldialogues.in
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Rhythm Pharma, Axovia Therapeutics Launch Joint Research Collaboration for Bardet-Biedl

Axfordia Therapeutics and Rhythm collaborate to advance understanding and treatment of Bardet-Biedl Syndrome (BBS), a rare ciliopathy affecting vision, obesity, and other conditions. Axfordia focuses on gene therapy for BBS, while Rhythm develops MC4R agonists like setmelanotide for hyperphagia and obesity in BBS patients.
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