First-ever authorized treatment option in the European Union for these rare genetic diseases of obesity
BOSTON, MA, USA I July 23, 2021 I 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 EU, IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above. IMCIVREE should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
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
IMCIVREE should be prescribed and supervised by physicians with expertise in obesity with underlying genetic etiology. IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:
- Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified 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 (polygenic) obesity.
See Full Product Information for IMCIVREEvi.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming 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 U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing and by the European Commission (EC) in July 2021 for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE is the first-ever FDA-approved and EC-authorized therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database – now with approximately 37,500 sequencing samples – to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company is based in Boston, MA.
i Huvenne H, Duberne B, Clément K, Poitou C. Rare genetic forms of obesity: clinical approach and current treatments in 2016. Obes Facts. 2016;9(3):158-173.
ii Ellacott KL, Cone RD. The role of the central melanocortin system in the regulation of food intake and energy homeostasis: lessons from mouse models. Philos Trans R Soc Land B Biol Sci. 2006;361(1471):1265-1274.
iii Ayers KL, Glicksberg BS, Garfield AS, et al. Melanocortin 4 receptor pathway dysfunction in obesity: patient stratification aimed at MC4R agonist treatment. J Clin Endocrinol Metab. 2018;103(7):2601-2612.
iv Krude H, Biebermann H, Luck W, Horn R, Brabant G, Grüters A. Severe early-onset obesity, adrenal insufficiency and red hair pigmentation caused by POMC variants in humans. Nat Genet. 1998;19(2):155-157.
v Clément, K., et al. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. The Lancet Diabetes & Endocrinology. Online first (2020). https://doi.org/10.1016/S2213-8587(20)30364-8
vi For the full product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.
SOURCE: Rhythm Pharmaceuticals
Post Views: 110
First-ever authorized treatment option in the European Union for these rare genetic diseases of obesity
BOSTON, MA, USA I July 23, 2021 I 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 EU, IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above. IMCIVREE should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.
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
IMCIVREE should be prescribed and supervised by physicians with expertise in obesity with underlying genetic etiology. IMCIVREE is not indicated for the treatment of patients with the following conditions as IMCIVREE would not be expected to be effective:
- Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified 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 (polygenic) obesity.
See Full Product Information for IMCIVREEvi.
About Rhythm Pharmaceuticals
Rhythm is a commercial-stage biopharmaceutical company committed to transforming 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 U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to POMC, PCSK1 or LEPR deficiency confirmed by genetic testing and by the European Commission (EC) in July 2021 for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. IMCIVREE is the first-ever FDA-approved and EC-authorized therapy for these rare genetic diseases of obesity. Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity. The Company is leveraging the Rhythm Engine and the largest known obesity DNA database – now with approximately 37,500 sequencing samples – to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. The company is based in Boston, MA.
i Huvenne H, Duberne B, Clément K, Poitou C. Rare genetic forms of obesity: clinical approach and current treatments in 2016. Obes Facts. 2016;9(3):158-173.
ii Ellacott KL, Cone RD. The role of the central melanocortin system in the regulation of food intake and energy homeostasis: lessons from mouse models. Philos Trans R Soc Land B Biol Sci. 2006;361(1471):1265-1274.
iii Ayers KL, Glicksberg BS, Garfield AS, et al. Melanocortin 4 receptor pathway dysfunction in obesity: patient stratification aimed at MC4R agonist treatment. J Clin Endocrinol Metab. 2018;103(7):2601-2612.
iv Krude H, Biebermann H, Luck W, Horn R, Brabant G, Grüters A. Severe early-onset obesity, adrenal insufficiency and red hair pigmentation caused by POMC variants in humans. Nat Genet. 1998;19(2):155-157.
v Clément, K., et al. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. The Lancet Diabetes & Endocrinology. Online first (2020). https://doi.org/10.1016/S2213-8587(20)30364-8
vi For the full product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.
SOURCE: Rhythm Pharmaceuticals
Post Views: 110
