Latest news with #POMC
Yahoo
06-05-2025
- Health
- Yahoo
Rhythm Pharmaceuticals Announces Participation in Upcoming Investor Conferences
Setmelanotide Indication In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). About Rhythm Pharmaceuticals Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. The fireside chats will be webcasted and available under 'Events & Presentations' in the Investor Relations section of the Company's website at and webcast replays will be available for 30 days following the presentations. BOSTON, May 06, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in fireside chats at three upcoming investor conferences: Story Continues In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide 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 BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the safety, efficacy, potential benefits of, and clinical design or progress of any of our products or product candidates at any dosage or in any indication, our participation in upcoming events and presentations, and the date, time and content thereof and the timing of any of the foregoing. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks, uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption 'Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact: David Connolly Head of Investor Relations and Corporate Communications Rhythm Pharmaceuticals, Inc. 857-264-4280 dconnolly@ Media Contact: Sheryl Seapy Real Chemistry (949) 903-4750 sseapy@
Yahoo
23-04-2025
- Business
- Yahoo
Rhythm Pharmaceuticals to Report First Quarter 2025 Financial Results on Wednesday, May 7, 2025
BOSTON, April 23, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that it will host a live conference call and webcast at 8:00 a.m. ET on Wednesday, May 7, 2025 to report its first quarter 2025 financial results and provide a corporate update. To access the live conference call, participants may register here. While not required, it is recommended that participants join the call ten minutes prior to the scheduled start. A webcast of the call will also be available under "Events and Presentations" in the Investor Relations section of the Rhythm Pharmaceuticals website at The archived webcast will be available on Rhythm Pharmaceuticals' website approximately two hours after the conference call and will be available for at least 30 days following the call. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide 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 BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the release of our financial results and our participation in upcoming events and presentations, including the timing of any of the foregoing. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks, uncertainties and other important factors, including those discussed under the caption 'Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Media Contact:Sheryl SeapyReal Chemistry(949) 903-4750sseapy@ Sign in to access your portfolio
Yahoo
17-03-2025
- Health
- Yahoo
'Dessert stomach' explains why you crave sweets after dinner — even if you're full
You've just devoured a big meal, where you've piled your plate high and are so full that you're considering unbuttoning your pants, and yet the moment someone mentions dessert, you're somehow willing and able to keep eating. It's like your regular stomach says, 'I'm stuffed,' but another part of your stomach chimes in, 'Hold my spoon — I've got this!' Turns out, there may be a scientific reason as to why this happens. It's called 'dessert stomach.' Now, researchers may have uncovered what causes it, and the reason may come as a surprise. Dessert stomach, aka a desire for sweets after a big meal, may not start in your stomach after all. Researchers have found that it actually starts in the brain. A recent study from the Max Planck Institute for Metabolism Research in Germany explored the impact of sugar consumption when we are already satiated. The findings pinpointed hypothalamic pro-opiomelanocortin (POMC) neurons, located in the hypothalamus (the brain region that regulates hormones and governs sensations like hunger, thirst, drowsiness and fullness), as being central to this phenomenon. Specifically, they discovered that the POMC neurons play a dual role. They help us feel full when we've eaten enough, but they also signal a desire for sugary treats afterward, which can lead to overeating. 'Research has shown that the same neurons that are responsible for signaling fullness also trigger cravings for sweets,' Dr. Britta Reierson, a family physician and obesity medicine specialist, tells Yahoo Life. It seems that even just thinking about or seeing sweets can activate these neurons, releasing a chemical called beta-endorphin that triggers feelings of pleasure. 'From an evolutionary perspective, this makes sense: Sugar is rare in nature, but provides quick energy. The brain is programmed to control the intake of sugar whenever it is available," explained Henning Fenselau, research group leader at the Max Planck Institute for Metabolism Research and head of the study, in a press release. 'This craving for dessert even when we are full is a fascinating phenomenon, and it involves a combination of factors,' says Reierson. 'Sugar naturally provides a rapid energy boost. So this drive to consume sugar, even when full, may have been advantageous for survival.' Reierson says there's also a relationship between pleasure and reward. 'The brain's reward system is activated by sugar, releasing pleasure-inducing chemicals that make sugary treats irresistible,' she explains. 'This craving can be even stronger when we perceive or taste dessert, enhancing that desire to eat even when we are full.' If you find dessert stomach coming on after enjoying a filling meal, here are a few creative ways to manage those cravings: 1. Do some strategic sweet swaps If your brain demands sugar, it's OK to give in to it — in moderation. A piece of dark chocolate can be a satisfying sweet that also gives your body a boost of antioxidants. Even a bowl of fresh berries or drizzling honey over Greek or plain yogurt can give you that satisfying sweet taste your body is craving. 2. Chew gum Chewing gum may help curb the desire for more food and help you feel satisfied. Data shows that the simple act of gum chewing for 45 minutes can help suppress cravings, thanks in part to the chewing motion, which signals to your brain a sense of satiety. Studies have also indicated that gum chewing stimulates the release of certain hormones associated with appetite control, making it a simple and effective tool to help manage hunger. 3. Try portion-controlled indulgence Serve yourself a minimal portion, such as a small scoop of ice cream or sorbet, and focus on savoring every bite. Eating slowly helps amplify enjoyment and signals your brain that you're satisfied with less. If you are dining with others, try sharing dessert with your table mates, so you can still enjoy a sweet treat without overdoing it. 4. Bake at home Baking at home provides the perfect opportunity to satisfy your sweet tooth while making healthier choices. For example, you can ensure that your treat doesn't contain high fructose corn syrup, and you add nutrient-dense foods like fruit and nuts. Experiment with recipes that include ingredients like Greek yogurt or fruit purees for added flavor and moisture without relying on unhealthy fats. Your dessert stomach may be hard to ignore sometimes, but with good choices and a little brain trickery, you can strike a sweet balance. Lauren Manaker is a dietitian and author.
CBC
13-03-2025
- Health
- CBC
7-year-old in Hamilton has rare condition with $300K annual treatment her family can't afford
Social Sharing Ibrahim Hermus arrived in Canada in 2023 with hopes his young daughter, Cena, would receive the medical care she desperately needed for a rare condition. But Ibrahim soon discovered that would not be the case. Cena, 7, was born in Turkey. At age 2, she was diagnosed with proopiomelanocortin (POMC) deficiency. The rare, life-threatening disorder causes severe obesity at an early age. According to the U.S. National Library of Medicine, people with the disorder have low levels of the hormone adrenocorticotropic (ACTH), which can come with other health-threatening complications. Cena and her family arrived in Canada on Sep. 12, 2023, as refugees from Turkey. Before that, they lived in Syria but had to flee the war-torn country. Ibrahim said Cena weighed around 165 pounds when they came to Canada, after deadly earthquakes hit both countries, and she's now around 220. During a recent visit to the family's Hamilton Mountain home, Ibrahim said he's "very scared" for his daughter. The treatment Cena needs — a medication called setmelanotide — is at least $300,000 during the first year, according to both the family and McMaster Children's Hospital, which has been giving care to the seven-year-old. CBC Hamilton has seen the hospital documents recommending the treatment for Cena. But that's a cost the family can't afford. Condition makes breathing, going to school hard At home, Cena has a shy demeanour and timid smile. She likes to hold onto a doll, which she says is her favourite, and loves playing with her little sister, Fatima. According to her father, Cena loves to spend time playing with her toys, as well as painting and drawing. But doing her favourite things can get difficult for Cena. Her condition makes her feel tired and unable to walk most of the time, and she's unable to attend school most days. Cena also suffers from sleep apnea, often waking up in the middle of the night, unable to breathe. Ibrahim said she's also dealing with diabetes, liver problems and constant pain. Along with obesity, people with POMC deficiency tend to have adrenal insufficiency, which can lead to periods of low blood sugar, seizures and high levels of a bilirubin, a toxic substance. Adrenal insufficiency can lead to death if not treated early, according to the U.S. National Library of Medicine. Health Canada says one in 12 people has a rare disease; many of them are children. CBC Hamilton reached out to the Canadian Organization for Rare Disorders to understand more about POMC deficiency in Canada, but did not receive a response before publication. The government of Canada has called POMC "very rare," citing the GlobalData Healthcare database. In the U.S., fewer than 1,000 people have the condition, according to Department of Health and Human Services. In Cena's case, the medication she needs is not covered by the Ontario Drug Benefit program. A spokesperson from the Ontario Ministry of Health told CBC Hamilton that for drugs like Imcivree (setmelanotide) to be covered under the program, the manufacturer must submit a request saying it is being used for a specific medical condition — and the ministry says the manufacturer has not done that. CBC Hamilton reached out to the manufacturer of Imcivree (setmelanotide) for comment but did not hear back before publication. The drug was approved by Health Canada for use here in 2023. 'My heart is broken for this family' Ibrahim said neither he nor his wife has been able to work in Canada due to a language barrier and the constant medical appointments and level of care their daughter needs. They get Ontario child benefit payments and food bank support from Mishka Social Services, an organization that helps the local Muslim community. Along with Cena, there are five other children — four at home who are two through 14 years old, and two adult children living in Canada. Over the past six months, Rania Mohammed, a social service worker with Mishka, has been working closely with the couple and their kids. "My heart is broken for this family," she told CBC Hamilton. She said this has been a difficult time for the family, especially Ibrahim, who feels "overwhelmed, scared, nervous" about what will happen to Cena. "He told me, 'Can you imagine the feeling when you see your loved one, my daughter, in front of me, and I cannot afford her medication? And I know if this won't be covered, I'm going to lose her,'" Mohammed said. Ibrahim said the anxiety has taken a big toll on him and his health, and he often finds it hard to sleep. Ozempic another option for Cena But Ibrahim won't stop trying to help his daughter, Mohammed said. Mishka Social Services has also been advocating for Cena. Since last year, the organization has set up a GoFundMe to raise money for her medication and is organizing a fundraising Iftar dinner during the holy month of Ramadan, with part of the proceeds going to help Cena, according to Mohammed. The dinner is Friday at Carmen's event centre. While setmelanotide remains out of reach right now, Cena is taking some medication to ease symptoms. Ozempic, a drug approved to treat diabetes, is also a temporary option to manage Cena's weight, Mohammed said. But even though it's less expensive — around $400 a month — the family says it does not fit in their budget. With help from Mishka and other services, Cena's family has reached out to multiple organizations and local officials such as members of Parliament to raise awareness of their situation and ask for "any support available." They have yet to receive any as a result, Mohammed said.
Yahoo
05-02-2025
- Business
- Yahoo
Rhythm Pharmaceuticals to Present at Oppenheimer Annual Healthcare Life Sciences Conference in February
BOSTON, Feb. 05, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will participate in a fireside chat at the upcoming Oppenheimer 35th Annual Healthcare Life Sciences Conference on Wednesday, February 12, 2025 at 8:40 a.m. ET. The fireside chat will be webcasted and available under 'Events & Presentations' in the Investor Relations section of the Company's website at A replay of the webcast will be available on the Rhythm website for 30 days following the presentation. About Rhythm PharmaceuticalsRhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm's lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm's headquarters is in Boston, MA. Setmelanotide IndicationIn the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide 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 BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity Contraindication Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression, suicidal ideation and depressed mood have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation, darkening of pre-existing nevi, development of new melanocytic nevi and increase in size of existing melanocytic nevi have occurred. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: IMCIVREE is not approved for use in neonates or infants. Serious and fatal adverse reactions including 'gasping syndrome' can occur in neonates and low birth weight infants treated with benzyl alcohol preserved drugs. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements our participation in upcoming events and presentations, including the timing thereof. Statements using words such as 'expect', 'anticipate', 'believe', 'may', 'will' and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, risks relating to our net revenues and anticipated financial results for the fiscal year ended December 31, 2024, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic, political and regulatory conditions, and the other important factors discussed under the caption 'Risk Factors' in Rhythm's Quarterly Report on Form 10-Q for the three months ended September 30, 2024 and other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise. Corporate Contact:David ConnollyHead of Investor Relations and Corporate CommunicationsRhythm Pharmaceuticals, Inc.857-264-4280dconnolly@ Media Contact:Sheryl SeapyReal Chemistry(949) 903-4750sseapy@ Effettua l'accesso per consultare il tuo portafoglio
