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First authorised treatment option in the European Union for these rare genetic diseases of obesity
Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial biopharmaceutical company committed to the transformation of the care of people living with rare genetic diseases of obesity, has announced today that the European Commission (CE) has granted the marketing authorization to IMCIVREE (setmelanotide) in the European Union (EU) for the treatment of obesity deficiency
“Rhythm phase 3 trials have confirmed that treatment with IMCIVREE could produce clinically significant effects on obesity and extreme hunger or hyperphagia. Many of the patients enrolled in these studies experienced an unprecedented weight loss 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 of the University of Ulm Medical Center in Germany. “With this authorization, we are reminded of the importance of genetic testing, in order to be able to properly identify and diagnose patients with POMC, PCSK1 or LEPR deficiency obesity and to provide eligible patients with BMI IVREE, 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 for patients from EU countries and other territories, including Northern Ireland, to address the underlying cause of obesities resulting from certain genetic defects in the melanocortin-4 receptor pathway (MC4),” said David Meeker, M.D., president and executive director of Rhythm. “This marks an important milestone for people in EU member states who live with POMC, PCSK1 or LEPR deficiency obesity, who can now have access to therapy that has proven to reduce hunger and body weight. We look forward to working closely with the EU-wide health authorities, while at the same time we started the country-by-country reimbursement process and work to make BMCIVREE available to eligible patients as soon as possible.”
Obesity due to POMC deficiency, PCSK1 or LEPR is an ultra rare disease caused by variants in genesPOMC, PCSK1orLEPRthat alter the pathway MC4R, which is a pathway in the hypothalamus responsible for regulating hunger, energy spending and, therefore, body weight.i,iiPeople who suffer obesity due to the deficiency of POMC, PCSK1 or LEPR struggle against extreme and insatiable hunger that begins at an early age, which results in a severe obesity of early genetic initiation.iii,iv
IMCIVREE EC authorization is based on the results of the major studies performed to date in obesity due to the deficiency of POMC, PCSK1 or LEPR.v In two phase 3 clinical trials, 80% of the ten patients with obesity due to POMC or PCSK1 deficiency achieved a body weight loss of more than 10% and 45.5% of the 11 patients with obesity due to LEPR deficiency achieved a body weight loss of more than 10% after a year of BMI IVREE treatment. In addition, in both studies, significant decreases were shown in the body mass index (BMI) in patients between 6 and 17 years of age at the beginning of the study (n=14).
In clinical trials, IMCIVREE was well tolerated in general. The most frequent adverse effects were reaction at the injection site, hyperpigmentation of the skin and nausea. Warnings and precautions include alterations in sexual excitement, depression and suicidal ideas, skin pigmentation and obscureness of pre-existing nevos.
IMCIVREE indicator (setmelanotide)vi
In the EU, IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with proopiomelanocortin deficiency (POMC) bialélic, including PCSK1, or the deficiency of the bialélic receptor (LEPR) with loss of genetically confirmed function in adults and children 6 years of age or older. IMCIVREE should be prescribed and supervised by a doctor with obesity experience with underlying genetic etiology.
In the United States, BMI is indicated for chronic weight control in adult and pediatric patients of 6 years of age or more with obesity due to proopiomelanocortin deficiency (POMC), proprotein convertasa subtilisine/kexine type 1 (PCSK1) or leptin receptor (LEPR). The condition must be confirmed by genetic tests that demonstrate variants in genesPOMC,PCSK1orLEPRwhich are interpreted as pathogenic, probably pathogenic or of uncertain importance (VOUS).
Limitations of usevi
IMCIVREE should be prescribed and supervised by doctors with obesity experience with underlying genetic etiology. BMIIVREE is not indicated for the treatment of patients with the following conditions, as BMI is not expected to be effective:
- Obesity due to suspected POMC, PCSK1 or LEPR deficiency with variantsPOMC,PCSK1orLEPRclassified as benign or likely benign;
- Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general obesity (polygenic).
Important security informationvi
WARNINGS AND PRECAUTIONS
Alteration of sexual excitation:Sexual adverse reactions may occur in patients treated with BMI IVREE. In clinical trials with BMI IVRE, spontaneous penis erections were produced in males and sexual adverse reactions in females. Tell patients who have an erection that lasts longer than 4 hours to seek emergency medical care.
Depression and suicidal ideas: In clinical trials, depression has been reported in patients treated with setmelanotide. Patients with depression should be supervised on each medical visit during treatment with BMIIVREE. The interruption of treatment with BMIVREE should be considered if patients experience suicidal thoughts or behaviors.
skin pigment and darkening of pre-existing nevus: setmelanotide can lead to a generalized increase in skin pigmentation and the darkening of pre-existing nevos due to its pharmacological effect. The skin tests of the entire body should be performed annually to monitor pigment lesions of the pre-existing and new skin before and during treatment with setmelanotide. Todos sobre leds e iluminación
Pediatric population:The use of BMI is not approved for newborns or infants. The safety and effectiveness of setmelanotide in children under the age of 6. No data available.
This medication contains 10 mg of bencyclic alcohol per ml. Bench alcohol can cause allergic reactions. Pregnant or breastfeeding patients should be warned of the potential risk of excipient bencyclic alcohol, which could accumulate over time and cause metabolic acidosis. This medication should be used with caution in patients with liver or kidney failure, due to the potential risk of excipient bencyclic alcohol that could accumulate over time and cause metabolic acidosis.
Adverse reactions
The most common adverse reactions (incidence ≥ 23 %) were reactions at the injection site, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection and spontaneous penis erection.
Use in specific populations:
Pregnancy
There is no data on the use of setmelanotide in pregnant women. Animal studies do not indicate direct adverse effects on reproductive toxicity. However, the administration of setmelanotide to pregnant conejas resulted in a decrease in maternal consumption of food, resulting in embryonic and fetal effects. As a precautionary measure, IMCIVREE should not start during pregnancy or while trying to become pregnant, as weight loss during pregnancy may result in fetal damage. If a patient taking setmelanotide has reached a stable weight and becomes pregnant, the maintenance of the treatment with setmelanotide should be considered as there was no evidence of teratogenicity in non-clinical data. If a patient who is taking setmelanotide and who still loses weight is pregnant, or the treatment with setmelanotide should be terminated, or the dose should be reduced while the patient is monitored to gain the recommended weight during pregnancy. The doctor responsible for the treatment should carefully monitor the weight during pregnancy in a patient taking setmelanotide.
Lactancy
It is unknown if the setmelanotide is excreted through human milk. A non-clinical study showed that setmelanotide is excreted through the milk of lactating rats. Measurable plasma setmelanotide concentrations of infants were not detected. A risk cannot be excluded for the newborn/lactant. A decision should be taken on whether to discontinue breastfeeding or to suspend/abstain therapy with BMIIVREE taking into account the benefit of breastfeeding for the child and the benefit of therapy for the mother.
Fertility
Human data on the effect of setmelanotide on fertility are not available. Animal studies did not indicate harmful effects on fertility
To report on SOSPECHAS OF WARNING REACTIONS, contact the national notification system that appears inhttps://www.ema.europa.eu/docs/en_GB/document_library/Template_or_form/2013/03/WC500139752.doc.
Consultationcomplete product informationfor IMCIVREEvi.
About Rhythm Pharmaceuticals
Rhythm is a commercial biopharmaceutical company committed to the transformation of the treatment paradigm for people living with rare genetic diseases of obesity. The company's precision medicine, IMCIVREE (setmelanotide), was approved in November 2020 by the United States Food and Drug Administration (FDA) for chronic weight control in adult and pediatric patients of 6 years of age and older with obesity due to POMC deficiency, PCSK1 or LEPR disease, confirmed by genetic impairment, and by 20 years of age. IMCIVREE is the first therapy approved by the FDA and authorized by the EC for these rare genetic diseases of obesity. Rhythm is enhancing a comprehensive clinical development program for setmelanotide in other rare genetic diseases of obesity. The company is taking advantage of the Rhythm engine and the largest known obesity DNA database, now with approximately 37,500 sequential samples, to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company has its headquarters in Boston, MA.
Corporate contact:
David Connolly
Director of Relations with Investors and Corporate Communications
Rhythm Pharmaceuticals, Inc.
857-264-4280
[email protected]
Contact from the investor:
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
[email protected]
Rhythm Pharmaceuticals announces the authorization of the European Commission of IMCIVREE® (setmelanotide) for the treatment of obesity and control of hunger associated with POMC deficiency, PCSK1 and LEPR
First authorised treatment option in the European Union for these rare genetic diseases of obesity /COMUNICAE/ /COMUNICAE/
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2024-05-20
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