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
Clinical Trials
Related News
epocrates.com
·

Imcivree now approved for younger children

FDA expanded Imcivree (setmelanotide) approval to children ≥2 years with obesity due to BBS or POMC, PCSK1, or LEPR deficiencies. A 52-week trial showed BMI reductions: -33.8% (POMC), -13.1% (LEPR), -9.7% (BBS). Common adverse events included skin hyperpigmentation and injection site reactions.

Related Clinical Trials:

Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
pharmabiz.com
·

Rhythm receives US FDA nod for IMCIVREE to reduce excess body weight & maintain

Rhythm Pharmaceuticals' IMCIVREE, approved by the FDA for children as young as 2, treats rare obesity causes like Bardet-Biedl syndrome and POMC, PCSK1, LEPR deficiencies by targeting the MC4R pathway, addressing hyperphagia and early-onset obesity.
medpagetoday.com
·

Imcivree Label Expanded; Weight Loss With Weekly Injection; Kidney Donation Chain

FDA expanded setmelanotide use for children with obesity. Bremelanotide showed promise in diabetic nephropathy. USPSTF advised against vitamin D and calcium for falls prevention. Cushing's syndrome patients have altered gut microbiomes. A new weight-loss injection outperformed others. Sexual-minority women reported better sexual health. Ohio State set a kidney donation record. Weight-loss drugs lead to other diagnoses. Palopegteriparatide now available in the U.S. Counterfeit GLP-1 drugs seized. Doctors advise on pausing GLP-1 drugs. WHO emphasizes obesity treatment beyond drugs. Demand for gender-affirming surgery rises.
worldpharmaceuticals.net
·

FDA expands Imcivree indication for children aged two and above

Rhythm Pharmaceuticals announces FDA approval of Imcivree for children aged 2 and above, targeting syndromic or monogenic obesity, including Bardet-Biedl syndrome and POMC/PCSK1/LEPR deficiencies. Imcivree is the first precision medicine for the MC4R pathway, showing significant weight and hunger reduction in trials.
stocktitan.net
·

Rhythm Pharma's IMCIVREE Wins FDA Approval for Rare Obesity Treatment in Young Children

Rhythm Pharmaceuticals announces FDA approval of IMCIVREE® (setmelanotide) for children as young as 2 years old with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or POMC, PCSK1, and LEPR deficiencies. IMCIVREE is the first precision medicine targeting MC4R pathway impairment, addressing hyperphagia and obesity in these rare conditions.
firstwordpharma.com
·

Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE (setmelanotide) for ...

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pharmacytimes.com
·

FDA Approves Expanded Indication for Setmelanotide to Include Patients 2 and Older

The FDA expanded setmelanotide (Imcivree) approval for children 2+ with syndromic or monogenic obesity due to Bardet-Biedl syndrome or POMC, PCSK1, LEPR deficiency, based on a phase 3 trial showing significant weight and hunger reduction. The treatment addresses insatiable hunger caused by MC4R pathway impairment, critical for early intervention to prevent long-term health complications.

Related Clinical Trials:

Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
morningstar.com
·

FDA Expands Rhythm Pharma Obesity Drug Approval to Younger Children

Rhythm Pharmaceuticals' weight-loss treatment, imcivree, received expanded FDA approval for children as young as two with obesity due to Bardet-Biedl syndrome or pro-opiomelanocortin. The drug, initially approved in 2020 for ages 6 and older, shows significant and sustained weight and hunger reduction.
investing.com
·

FDA expands approval for obesity drug to include young children

The FDA expanded Rhythm Pharmaceuticals' obesity drug IMCIVREE's indication to include children as young as 2, targeting MC4R pathway diseases. The approval is based on the drug's ability to reduce excess body weight and maintain long-term weight reduction, though it comes with side effects. Rhythm Pharmaceuticals has seen a stock surge and maintains strong liquidity.
morningstar.com
·

Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide)

Rhythm Pharmaceuticals announces FDA approval of IMCIVREE for children as young as 2 with syndromic or monogenic obesity due to Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiency, targeting hyperphagia and obesity via the MC4R pathway.
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