Rhythm Pharmaceuticals Announces European Commission Authorization of IMCIVREE® (setmelanotide)? for the Treatment of Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency
BOSTON, July 23, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced that the European Commission (EC) has granted marketing authorization to IMCIVREE (setmelanotide) in the European Union (EU) for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.
“Rhythm’s Phase 3 trials confirmed that treatment with IMCIVREE may deliver clinically meaningful impacts on obesity and severe hunger or hyperphagia. Many patients enrolled in these studies experienced weight loss of a magnitude that is unprecedented in the natural history of rare genetic diseases of obesity,” said Martin Wabitsch, M.D., professor of medicine and head of the Division of Pediatric Endocrinology and Diabetes at Ulm University Medical Center in Germany. “With this authorization, we are reminded of the importance of genetic testing, so that we can identify and properly diagnose patients with POMC, PCSK1 or LEPR deficiency obesity and offer eligible patients IMCIVREE, a pharmacological therapy designed to address the underlying cause of their disease.”
“With this authorization now in the EU, IMCIVREE becomes the first and only treatment option available to patients in EU countries and other territories including Northern Ireland to address the underlying cause of obesities driven by certain genetic defects in the melanocortin-4 (MC4) receptor pathway,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “This marks an important milestone for people in the EU member states living with POMC, PCSK1 or LEPR deficiency obesities, who now may have access to a therapy that has been shown to reduce hunger and body weight. We look forward to working closely with health authorities throughout the EU, as we commence the country-by-country reimbursement process and work to make IMCIVREE available to eligible patients as rapidly as possible.”
Obesity due to POMC, PCSK1 or LEPR deficiency is an ultra-rare disease caused by variants in POMC, PCSK1 or LEPR genes that impair the MC4R pathway, which is a pathway in the hypothalamus that is responsible for regulating hunger, energy expenditure and consequently body weight.i,ii People living with obesity due to POMC, PCSK1 or LEPR deficiency struggle with extreme, insatiable hunger beginning at a young age, resulting in early-onset, severe obesity.iii,iv As an MC4R agonist, IMCIVREE is designed to restore impaired MC4R pathway activity arising due to genetic deficits upstream of the MC4 receptor.
The EC authorization of IMCIVREE is based on results from the largest studies conducted to date in obesity due to POMC, PCSK1 or LEPR deficiency.v In two Phase 3 clinical trials, 80 percent of ten patients with obesity due to POMC or PCSK1 deficiency achieved greater than ten percent body weight loss and 45.5 percent of 11 patients with obesity due to LEPR deficiency achieved greater than 10 percent body weight loss after one year of treatment with IMCIVREE. Additionally, in both studies, significant decreases in body mass index (BMI) were demonstrated across patients who were 6 to 17 years old at baseline (n=14).
In clinical trials, IMCIVREE was generally well-tolerated. The most common adverse events were injection site reaction, skin hyperpigmentation and nausea. Warnings and precautions include disturbance in sexual arousal, depression and suicidal ideation, skin pigmentation and darkening of pre-existing nevi.
IMCIVREE (setmelanotide) Indicationvi
In the United States, IMCIVREE is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. The condition must be confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VOUS).
Limitations of Usevi
Important Safety Informationvi
WARNINGS AN PRECAUTIONS
Disturbance in Sexual Arousal: Sexual adverse reactions may occur in patients treated with IMCIVREE. Spontaneous penile erections in males and sexual adverse reactions in females occurred in clinical studies with IMCIVREE. Instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention.
Depression and Suicidal Ideation: In clinical trials, depression has been reported in patients treated with setmelanotide. Patients with depression should be monitored at each medical visit during treatment with IMCIVREE. Consideration should be given to discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors.
Skin Pigmentation and Darkening of Pre-Existing Nevi: Setmelanotide may lead to generalized increased skin pigmentation and darkening of pre-existing nevi because of its pharmacologic effect. Full body skin examinations should be conducted annually to monitor pre-existing and new skin pigmentary lesions before and during treatment with setmelanotide.
Pediatric Population: IMCIVREE is not approved for use in neonates or infants. The safety and efficacy of setmelanotide in children less than 6 years of age has not yet been established. No data are available.
This medicinal product contains 10 mg benzyl alcohol in each ml. Benzyl alcohol may cause allergic reactions. Patients who are pregnant or breastfeeding should be advised of the potential risk from the excipient benzyl alcohol, which might accumulate over time and cause metabolic acidosis. This medicinal product should be used with caution in patients with hepatic or renal impairment, because of the potential risk from the excipient benzyl alcohol which might accumulate over time and cause metabolic acidosis.
USE IN SPECIFIC POPULATIONS
To report SUSPECTED ADVERSE REACTIONS, contact the national reporting system listed in http://www.ema.europa.eu/docs/en_GB/document_library/Template_or_form/2013/03/WC500139752.doc.
See Full Product Information for IMCIVREEvi.
About Rhythm Pharmaceuticals
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