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Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial biopharmaceutical company committed to the transformation of care to people with rare genetic diseases of obesity, has announced today that it has submitted its application to the European Medicines Agency (EMA) for the IMCIVREE® Type II variation (setmelanotide)
Ryth Pharmaceuticals has announced the submission of the application to the European Medicines Agency (EMA) for the type II variation of IMCIVREE® (setmelanotide) for the treatment of obesity and control of hunger in adult and pediatric patients of six years of age or more with Bardet-Biedl syndrome (BBS) or Alström syndrome.
“This marks an important milestone towards our goal of bringing BBS patients worldwide or with many other rare genetic diseases of obesity,” said David Meeker, PhD in Medicine, President and Executive Director of Rhythm. “IMCIVREE achieved clinical and statistically significant results in our phase 3 trial in BBS, and demonstrated a marked and sustained weight loss in patients with Alström syndrome treated in our phase 2 and 3 trials. Based on this data, we believe that BMCIVREE will be the first medication to effectively address the early-initiated severe obesity and hyperphagia that characterize these diseases. We look forward to working closely with the regulatory authorities of the European Union to bring BMCIVREE to these additional populations.2
“The BBS community in the EU is particularly well defined, with more than 1500 patients diagnosed and treated in academic centers,” said Yann Mazabraud, executive vice president and director of the international area in Rhythm. “It should be noted that many of these patients have severe obesity and hyperphagia for which treatment with BMI has been conceived. We are eager to continue working to improve BBS knowledge and the potential benefits of BMCIVREE and, if authorized, we hope to launch this treatment to the market quickly as a key step in transforming the attention of people with rare genetic diseases of obesity.”
The presentation to the EMA is based on the data of the Rhythm phase 3 critical clinical trial on the setmelanotide in patients with BBS or Alström syndrome. As previously reported, the study met its primary valuation criteria and all key secondary valuation criteria, with statistically and clinically significant weight and hunger reductions at 52 weeks of treatment. All patients who met the primary valuation criterion defined as a weight loss of over 10% had BBS and none had Alström syndrome. However, the data in this phase 3 test are backed by the results of the phase 2 test, which suggest that the treatment with setmelanotide can lead to a decrease in weight and hunger in people with Alström syndrome. In addition, data from a predefined exploratory valuation criterion showed that, in patients with BBS and Alström syndrome under the age of 18, treatment with setmelanotide was associated with clinically significant reductions in IMC-Z scores. The IMC-Z score, or standard BMI deviation score, represents the number of standard deviations regarding the median BMI by age and sex of the child.
About Bardet-Biedl and Alström syndromes
BBS and Alström syndrome are very rare genetic diseases that affect multiple organ systems. The clinical characteristics of BBS may include cognitive impairment, polydactilia, renal dysfunction, hypogonadism and visual dysfunction. The clinical characteristics of Alström syndrome may include progressive visual and hearing impairment, insulin resistance and type 2 diabetes, hyperlipidemia, progressive renal dysfunction, myocardiopathy and short stature in adulthood. Insatiable hunger, also known as hyperphagia, and early severe obesity are common in people with BBS or Alström syndrome. Rhythm estimates that BBS affects approximately 1500 to 2500 people and that Alström syndrome affects approximately 500 people in the United States, with a similar prevalence estimate in Europe. Currently, there are no approved therapies aimed at MC4R to reduce body weight and hunger in BBS or Alström syndrome.
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 Precision Medicine of Rhythm, IMCIVREE (setmelanotide), was approved in November 2020 by the United States Food and Drug Administration (FDA, by its acronyms in English) for chronic weight control in adult patients and pediatrics of 6 years of age or more with obesity due to POMC, PCSK1 or LEPR deficiency IMCIVREE is the first therapy approved by the FDA and authorized by the EC and MHRA for patients with these rare genetic diseases of obesity. Rhythm is making progress in a comprehensive setmelanotide clinical development program in other rare genetic diseases of obesity, and is taking advantage of the resources of the Rhythm Engine and the largest known DNA database of obesity, with approximately 37,500 sequential samples, to improve the understanding, diagnosis and care of people suffering from severe obesity due to certain genetic deficiencies. Rhythm headquarters is located in Boston, Massachusetts. Cómo adoptar un perro en Verín
IMCIVREE indicator® (setmelanotida)
In the EU and in Britain, BMIIVREE is indicated for the treatment of obesity and the control of hunger associated with bialélic POMC deficiency, including PCSK1, or bialélic LEPR deficiency with genetically confirmed loss of 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 genes POMC, PCSK1 or LEPR which are interpreted as pathogenic, probably pathogenic or of uncertain importance (VUS).
Important security information
Warnings and precautions:
Alteration of sexual excitation: Sexual adverse reactions may occur in patients treated with BMI IVREE. In clinical trials with BMI IVREE, 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: Some medicines that address the central nervous system, such as BMI, can cause depression or suicidal ideas. Watch patients for a new beginning or worsening of depression. Consider discontinuing treatment with BMI IVREE if patients experience suicidal thoughts or behaviors.
Pigmentation of the skin and darkening of pre-existing nevos: IMCIVREE can cause a widespread increase in skin pigmentation and the darkening of pre-existing nevos due to its pharmacological effect. This effect is reversible after the interruption of the drug. Perform an entire body skin test before the start and periodically during treatment with BMIIVREE to monitor new and pre-existing skin pigment lesions.
Risk of severe adverse reactions due to the conservant of bencyclic alcohol in neonates and low-weight infants at birth: The use of BMI is not approved for newborns or infants.
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
It disrupts treatment with BMI when pregnancy is detected, unless the benefits of therapy exceed the potential risks to the fetus.
The use of BMI IVREE treatment is not recommended during breastfeeding.
To report suspicions of adverse reactions, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 orwww.fda.gov/medwatch.
Consultation Information for full recipes and Technical data of the EU for IMCIVREE.
Invalid request: request (2174) exceeds text limit (2000)Rhythm Pharmaceuticals completes the presentation of the application for type II variation to the European Medicine Agency for IMCIVREE® (setmelanotide) for Bardet-Biedl and Alström syndromes
Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial biopharmaceutical company committed to the transformation of care to people with rare genetic disease
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2023-04-11
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