This section highlights FDA-related milestones and regulatory updates for drugs developed by Rhythm Pharmaceuticals (RYTM).
Over the past two years, Rhythm Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Setmelanotide, IMCIVREE, bivamelagon, and LB54640. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Setmelanotide (HO) FDA Regulatory Timeline and Events
Setmelanotide (HO) is a drug developed by Rhythm Pharmaceuticals for the following indication: Hypothalamic Obesity.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Setmelanotide (HO)
- Announced Date:
- May 12, 2026
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced that Rhythm and its collaborators will present six posters, including four featuring new data, relating to the company's work with setmelanotide at The European Congress of Endocrinology (ECE), taking place May 9-12, 2026 in Prague.
AI Summary
Rhythm Pharmaceuticals said it and its collaborators will present six posters, including four with new data, at the European Congress of Endocrinology in Prague from May 9-12, 2026. The presentations focus on setmelanotide and its effects in rare obesity-related conditions. One real-world study in France found that 62 adults with acquired hypothalamic obesity treated for up to 12 months had meaningful drops in BMI and hunger scores. Another analysis from the Phase 3 TRANSCEND trial showed improvements in several cardiometabolic risk measures compared with placebo.
Other research will look at how severe hyperphagia, or extreme hunger, is in adults with Bardet-Biedl syndrome in the UK and show that it may be underreported in surveys. A separate study will highlight how acquired hypothalamic obesity remains a serious but often overlooked burden for people with childhood-onset craniopharyngioma across different life stages. Two encore posters will also be shown. Together, the data support the possible benefits of setmelanotide and the need for better recognition of these rare conditions.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- May 1, 2026
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced the European Commission (EC) has expanded the marketing authorization for IMCIVREE® (setmelanotide) to include the treatment of obesity and control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity (HO) due to hypothalamic injury or impairment.
AI Summary
Rhythm Pharmaceuticals announced the European Commission expanded the marketing authorization for IMCIVREE (setmelanotide) to treat obesity and control hunger in adults and children aged 4 years and older with acquired hypothalamic obesity caused by hypothalamic injury or impairment. Acquired HO is a rare neuroendocrine disease that causes rapid, sustained weight gain; Rhythm estimates about 10,000 people in Europe have acquired HO.
With EU authorization in place, Rhythm will work to secure access on a country-by-country basis across Europe and anticipates commercial launches in 2027. IMCIVREE is limited to specific patient groups and is not indicated for obesity from benign POMC, PCSK1, or LEPR variants, other genetic syndromes, Bardet‑Biedl syndrome, or general polygenic obesity.
Important safety notes: IMCIVREE is contraindicated in patients with prior serious hypersensitivity. Warnings include changes in sexual arousal (spontaneous erections), depression and suicidal ideation, hypersensitivity reactions, skin hyperpigmentation and new or darkened nevi, and risk of acute adrenal insufficiency in some patients. Common side effects include skin darkening, injection site reactions, nausea, headache, gastrointestinal symptoms, vomiting, and depression. Not recommended during breastfeeding; see full prescribing information for details.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- March 19, 2026
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for IMCIVREE® (setmelanotide) to treat patients living with acquired hypothalamic obesity (HO).
AI Summary
Rhythm Pharmaceuticals announced that the U.S. Food and Drug Administration has approved an expanded indication for IMCIVREE® (setmelanotide) to treat acquired hypothalamic obesity (HO). It is the first and only FDA-approved therapy for acquired HO and is indicated to reduce excess body weight and maintain weight loss long term in adults and pediatric patients aged 4 years and older.
Acquired HO is a rare condition of accelerated, sustained weight gain caused by hypothalamic injury or dysfunction, often following tumors or their treatment. Rhythm estimates about 10,000 people in the U.S. have acquired HO. The approval was supported by the global Phase 3 TRANSCEND trial (N=142), in which setmelanotide achieved a placebo-adjusted BMI reduction of 18.4%.
Rhythm will host a conference call today at 7:00 p.m. ET to discuss the approval. IMCIVREE is not indicated for other types of obesity; important safety considerations include risk of serious hypersensitivity, depression and suicidal ideation, skin hyperpigmentation, and possible adrenal insufficiency. Common adverse reactions include injection site reactions, nausea, headache, and gastrointestinal symptoms.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- March 1, 2026
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced additional positive data from its global Phase 3 TRANSCEND trial of setmelanotide in patients with acquired hypothalamic obesity (HO).
AI Summary
Rhythm Pharmaceuticals announced additional positive results from its global Phase 3 TRANSCEND trial of setmelanotide in people with acquired hypothalamic obesity. At 52 weeks, the drug produced an 18.8% placebo-adjusted reduction in BMI in the full study group (N = 142). The analysis included 12 Japanese patients and 10 supplemental patients with acquired hypothalamic obesity, and shows sustained weight benefit over one year compared with placebo.
The 52-week data also reported safety findings. Common adverse events included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erections in males. The company highlighted the need to monitor mood changes and skin lesions during treatment. Overall, the results support continued development of setmelanotide as a potential treatment option for acquired hypothalamic obesity.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- December 11, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced positive preliminary results from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS).
AI Summary
Rhythm Pharmaceuticals reported positive preliminary results from an exploratory Phase 2 trial of setmelanotide in people with Prader-Willi syndrome (PWS) and said it plans a Phase 3 registrational trial pending completion of Phase 2. The open-label study enrolled 18 patients aged 6–65 with obesity and was extended to 52 weeks. Seventeen of 18 participants remain on therapy.
As of the Nov. 14 cut-off, BMI and hyperphagia improvements were seen at Month 3 (n=8) and Month 6 (n=5). Six of eight patients at Month 3 had BMI reductions. Three of five at Month 6 had BMI decreases (two larger than at Month 3, one unchanged). Six of seven evaluable patients at Month 3 showed meaningful reductions on the Hyperphagia Questionnaire for Clinical Trials; one patient had a baseline score of zero and was not evaluable.
Safety and tolerability were consistent with the drug’s known profile. Rhythm also started a Part D arm of a Phase 1 study of weekly MC4R agonist RM-718 in PWS, planning to screen the first patients in December 2025.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- December 10, 2025
- Target Action Date:
- December 11, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced that it will host a live conference call and webcast on Thursday, December 11 at 8:00 a.m. ET to disclose preliminary data from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS).
AI Summary
Rhythm Pharmaceuticals announced it will host a live conference call and webcast on Thursday, December 11 at 8:00 a.m. ET to disclose preliminary data from its exploratory Phase 2 trial of setmelanotide in patients with Prader‑Willi syndrome (PWS). The company said the call will focus on early findings from the study, which is evaluating setmelanotide for symptoms linked to PWS, and that management will discuss the data and next steps with listeners.
Participants may register for the conference call on Rhythm’s investor relations site. The company recommends joining the call ten minutes before the scheduled start. A live webcast will be available under “Events and Presentations” at https://ir.rhythmtx.com/. An archived recording will appear about two hours after the call and remain available for 30 days.
Rhythm is a biopharmaceutical company developing treatments for rare neuroendocrine diseases and is sharing this preliminary PWS data as part of its ongoing clinical program.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- November 10, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced that Rhythm and its partners delivered four Rhythm data presentations at ObesityWeek® 2025, held last week in Atlanta, GA.
AI Summary
Rhythm Pharmaceuticals and partners presented four data sets at ObesityWeek® 2025 in Atlanta that highlight setmelanotide’s effects in acquired hypothalamic obesity. Overall, the findings showed large, sustained BMI reductions, meaningful improvements in cardiometabolic health, and positive patient- and caregiver-reported outcomes, suggesting the drug may address both weight and daily functioning in this severe condition.
Key presentations included a post-hoc analysis of the Phase 3 TRANSCEND trial showing a 27.1% mean placebo-adjusted BMI reduction for patients (n=9) treated with setmelanotide plus GLP-1 therapy versus GLP-1 plus placebo (n=6), and a 19.0% placebo-adjusted BMI reduction for setmelanotide-treated patients (n=72) versus placebo (n=33). A second TRANSCEND poster reported improvements across blood pressure, lipids, and proteomic biomarkers after 52 weeks. A patient- and caregiver-experience poster reported reduced hunger and better energy and activity. A separate study of 212 children explored hyperphagia and genetic variants linked to early-onset obesity.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- August 20, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the company's supplemental New Drug Application (sNDA) for setmelanotide seeking approval for the treatment of conditions associated with acquired hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals announced that the U.S. Food and Drug Administration has accepted for filing its supplemental New Drug Application (sNDA) for setmelanotide to treat conditions linked to acquired hypothalamic obesity. The FDA granted Priority Review status and set a PDUFA goal date of December 20, 2025, meaning it aims to complete its review by that date.
In Europe, the European Medicines Agency validated a Type II variation to the marketing authorization for setmelanotide, starting its review on August 16, 2025. If approved, it would be the first treatment for patients with obesity caused by hypothalamic injury, which often leads to reduced energy use and increased hunger.
The filing is supported by Phase 3 TRANSCEND trial data showing about a 20% placebo-adjusted BMI reduction over 52 weeks. Rhythm will host an investor event in Boston on September 24 to discuss commercial plans.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- July 12, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced data from three new presentations on the Company's clinical programs for acquired hypothalamic obesity at the Endocrine Society's Annual Meeting (ENDO 2025) taking place July 12-15 in San Francisco, CA.
AI Summary
Rhythm Pharmaceuticals recently shared promising new data at ENDO 2025 in San Francisco for its clinical programs targeting acquired hypothalamic obesity. The company presented three new studies focusing on reducing body mass index (BMI) through novel treatments. One key presentation detailed the Phase 3 TRANSCEND trial of setmelanotide, which achieved a 19.8% placebo-adjusted difference in BMI reduction across different age and sex groups. Notably, participants who had used or were using GLP-1 therapies experienced BMI reductions of up to 25.1% at 52 weeks. Another study on a new oral MC4R agonist, bivamelagon, showed statistically significant BMI decreases in patients over a 14-week period. A third presentation shared qualitative insights from patients and caregivers, highlighting the real-life impact of the condition and potential benefits of these targeted treatments.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- June 25, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, announced that three late-breaking abstracts have been accepted for presentation at The Endocrine Society's Annual Meeting (ENDO 2025) taking place July 12-15 in San Francisco, CA.
AI Summary
Rhythm Pharmaceuticals announced that three late-breaking abstracts have been accepted for presentation at The Endocrine Society’s Annual Meeting (ENDO 2025) in San Francisco, CA, from July 12-15. This significant event will feature cutting-edge research on treatments for acquired hypothalamic obesity. One highlight is a live oral presentation by Dr. Susan Phillips from the University of California San Diego/Rady Children’s Hospital, who will share data from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide. In addition, Dr. Christian Roth from Seattle Children’s Research Institute and Dr. Vidhu Thaker from Columbia University will present posters that detail patient exit interviews and results from a Phase 2 trial of an oral MC4R agonist, bivamelagon, respectively.
The presentations aim to strengthen understanding of novel therapeutic strategies for managing obesity linked to neuroendocrine disorders.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- May 14, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, announced the presentation of new, real-world data at two European congresses that show consistent improvements in body mass index (BMI), BMI-z, and hunger scores in a total of 35 patients with acquired or congenital hypothalamic obesity who were treated with setmelanotide for up to nine months.
AI Summary
Rhythm Pharmaceuticals recently presented new real-world data from two European congresses showing that setmelanotide therapy led to consistent improvements in patients with hypothalamic obesity. The study involved 35 patients—30 with acquired and five with congenital hypothalamic obesity—who received treatment for up to nine months. Results revealed meaningful reductions in body mass index (BMI) for adults and decreases in BMI-z scores for pediatric patients, with improvements noted as early as three months and deepening at six and nine months. Along with the weight loss benefits, patients also experienced notable reductions in hunger scores. These findings support earlier clinical trial data and underscore the potential of setmelanotide, an MC4R agonist, to address the significant challenges of weight gain and overwhelming hunger in both acquired and congenital hypothalamic obesity.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- April 7, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced positive topline results from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, a melanocortin-4 receptor (MC4R) agonist, for the treatment of acquired hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals, Inc. announced positive topline results from its pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, a melanocortin-4 receptor (MC4R) agonist, for treating acquired hypothalamic obesity. The trial showed that patients on setmelanotide experienced a mean BMI reduction of 16.5% at 52 weeks, compared to a 3.3% increase in the placebo group (p<0.0001). Adult patients achieved a 19.2% placebo-adjusted BMI reduction, while those under 18 reached a 20.2% reduction.
The results indicate statistically significant and clinically meaningful improvements for both adult and pediatric patients suffering from this challenging condition. With no new safety concerns observed, the promising data provide hope for a targeted treatment option for acquired hypothalamic obesity. Rhythm Pharmaceuticals plans to submit regulatory applications in the U.S. and EU by the third quarter of 2025.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- April 6, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced the Company will hold a conference call and webcast on Monday, April 7, 2025 at 8:00 a.m. ET to disclose topline results from the Pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, a melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals, Inc. announced a conference call and webcast on Monday, April 7, 2025 at 8:00 a.m. ET. During this call, the company will reveal topline results from its Pivotal Phase 3 TRANSCEND trial. This trial examines the safety and efficacy of setmelanotide, a melanocortin-4 receptor (MC4R) agonist, in patients suffering from acquired hypothalamic obesity—a condition that arises after damage to the hypothalamus, often following brain surgery or injury.
The TRANSCEND trial is a global, randomized, double-blind, placebo-controlled study involving patients aged 4 years and older. Its primary focus is to assess the mean percent change in body mass index (BMI) from baseline after 52 weeks of treatment, while secondary measures include changes in hunger levels, weight, and overall quality of life. Interested parties can join the conference call or view the webcast through Rhythm Pharmaceuticals’ Investor Relations website.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- March 19, 2025
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced it has received orphan drug designation from Japan's Ministry of Health, Labour and Welfare (MHLW) for setmelanotide as a treatment for acquired hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals, Inc. has received orphan drug designation from Japan’s Ministry of Health, Labour and Welfare for setmelanotide as a treatment for acquired hypothalamic obesity. This rare condition, often caused by injuries to the hypothalamus from brain tumors or trauma, leads to rapid and sustained weight gain along with extreme hunger. The designation highlights Japan’s recognition of the high unmet medical need for safe and effective treatments for this debilitating disease.
Setmelanotide, a melanocortin-4 receptor (MC4R) agonist, is being evaluated in a global Phase 3 trial, with top-line results anticipated in the second quarter of 2025. With orphan drug status granted in both Japan and Europe, Rhythm is well positioned to advance its global strategy and potentially provide the first treatment that targets the underlying biology of acquired hypothalamic obesity.
Read Announcement - Drug:
- Setmelanotide (HO)
- Announced Date:
- November 18, 2024
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced the presentation of new, real-world data that showed four pediatric patients with acquired hypothalamic obesity or congenital hypothalamic obesity achieved >5% weight reduction at three months on setmelanotide, a melanocortin-4 receptor (MC4R) agonist.
AI Summary
Rhythm Pharmaceuticals shared new real-world data showing that four pediatric patients with hypothalamic obesity achieved significant weight loss on setmelanotide therapy. In this French early-access program, two children with congenital hypothalamic obesity and two with acquired hypothalamic obesity experienced more than a 5% reduction in body weight within three months of starting treatment. Setmelanotide, an MC4R agonist, targets the disrupted pathway that contributes to obesity in these patients. The findings support the potential of setmelanotide to address the underlying cause of obesity when traditional lifestyle changes and standard treatments may not work effectively. Building on these encouraging results, Rhythm also announced plans to launch a 34-week substudy to evaluate the treatment in 39 patients with congenital hypothalamic obesity, further exploring its benefits for younger patients facing this challenging condition.
Read Announcement- Drug:
- Setmelanotide (HO)
- Announced Date:
- May 22, 2024
- Indication:
- Hypothalamic Obesity
Announcement
Rhythm Pharmaceuticals, Inc. announced the National Institute for Health and Care Excellence (NICE) has issued guidance that recommends IMCIVREE® (setmelanotide) as an option for treating obesity and the control of hunger (hyperphagia) in genetically confirmed Bardet-Biedl syndrome (BBS) in people 6 years of age and over, if they are between 6 and 17 years of age when treatment starts. Patients may remain on reimbursed setmelanotide as adults whilst they continue to benefit from therapy.
AI Summary
Rhythm Pharmaceuticals announced that the National Institute for Health and Care Excellence (NICE) has recommended IMCIVREE® (setmelanotide) for treating obesity and controlling hunger (hyperphagia) in patients with genetically confirmed Bardet-Biedl syndrome (BBS). The guidance specifies that the treatment is for individuals 6 years and older, provided that therapy begins between the ages of 6 and 17. Importantly, patients who see benefits from the treatment as children may continue to receive reimbursed setmelanotide into adulthood. This endorsement by NICE marks a key step in making targeted treatment available to those with BBS in England and Wales. It also reflects the collaborative efforts between leading clinical experts, advocacy groups, and NICE to address the serious challenges of hyperphagia and severe obesity associated with BBS, offering hope for improved quality of life for affected families.
Read Announcement
IMCIVREE (setmelanotide) FDA Regulatory Timeline and Events
IMCIVREE (setmelanotide) is a drug developed by Rhythm Pharmaceuticals for the following indication: Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency.
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- IMCIVREE (setmelanotide)
- Announced Date:
- March 26, 2026
- Indication:
- Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency
Announcement
Rhythm Pharmaceuticals, Inc. nnounced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending to expand the current marketing authorization for
AI Summary
Rhythm Pharmaceuticals announced that the European Medicines Agency’s CHMP has adopted a positive opinion recommending an expansion of the current marketing authorization for setmelanotide (IMCIVREE). The opinion will be reviewed by the European Commission, with a decision expected in the second quarter of 2026. If approved, the change would alter IMCIVREE’s allowable uses in the EU.
The recommendation includes limits: setmelanotide is not indicated for obesity from POMC, PCSK1, or LEPR variants classified as benign or likely benign, nor for obesity not related to acquired HO, Bardet–Biedl syndrome, or those gene deficiencies. Key safety points: contraindicated in patients with prior serious hypersensitivity; warnings for spontaneous penile erections/priapism, depression and suicidal ideation, hypersensitivity reactions, and acute adrenal insufficiency in acquired HO. Common adverse reactions include skin hyperpigmentation, injection site reactions, nausea, headache, GI symptoms, vomiting, depression, and penile erection. Avoid breastfeeding and discontinue in pregnancy unless benefits outweigh risks.
Read Announcement- Drug:
- IMCIVREE (setmelanotide)
- Announced Date:
- November 7, 2025
- Indication:
- Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency
Announcement
Rhythm Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has extended by three months the review period for the supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide) for the treatment of acquired hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals said the FDA extended the review period for its supplemental New Drug Application (sNDA) of IMCIVREE® (setmelanotide) for acquired hypothalamic obesity by three months, moving the PDUFA goal date from December 20, 2025, to March 20, 2026.
The FDA requested additional sensitivity analyses of clinical efficacy data from the Phase 3 trial. No new data or safety and manufacturing information were requested. This major amendment grants the agency extra review time without raising new safety or production questions.
Acquired hypothalamic obesity is a rare condition with no approved treatments, caused by hypothalamic damage. Rhythm’s CEO David Meeker, MD, said the analyses will confirm IMCIVREE’s potential to help patients without current options. The company remains in close contact with the FDA and is preparing to provide treatment. A live conference call and webcast will start today at 8:00 a.m. ET.
Read Announcement- Drug:
- IMCIVREE (setmelanotide)
- Announced Date:
- December 20, 2024
- Indication:
- Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency
Announcement
Rhythm Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for IMCIVREE® (setmelanotide) to include children as young as 2 years old.
AI Summary
Rhythm Pharmaceuticals announced that the FDA has expanded the use of its drug IMCIVREE® (setmelanotide) to include children as young as 2 years old. This new indication covers pediatric patients with syndromic or monogenic obesity caused by Bardet-Biedl syndrome (BBS) or genetic deficiencies in pro-opiomelanocortin (POMC), PCSK1, or the leptin receptor (LEPR). These conditions affect the melanocortin-4 receptor (MC4R) pathway, leading to insatiable hunger and early-onset obesity.
This targeted treatment is the first precision medicine aimed at addressing the root cause of abnormal eating behaviors in patients with these rare diseases. By reducing excess body weight and maintaining weight reduction over time, IMCIVREE offers a much-needed option for young children struggling with severe obesity and related health issues. Early intervention with this approved therapy has the potential to lessen long-term complications associated with these conditions.
Read Announcement- Drug:
- IMCIVREE (setmelanotide)
- Announced Date:
- December 3, 2024
- Indication:
- Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency
Announcement
Rhythm Pharmaceuticals, Inc. announced that the United Kingdom's Medicines & Healthcare products Regulatory Agency (MHRA) has expanded the marketing authorization for IMCIVREE® (setmelanotide) to include the treatment of obesity and control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) or 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 adult and pediatric patients as young as 2 years old and older.
AI Summary
Rhythm Pharmaceuticals announced that the UK’s MHRA has expanded the marketing authorization for IMCIVREE (setmelanotide). Under the new authorization, the treatment is approved for managing obesity and controlling hunger in patients with genetically confirmed Bardet-Biedl syndrome (BBS) or loss-of-function mutations in pro-opiomelanocortin (POMC), including deficiencies in PCSK1 or the leptin receptor (LEPR).
This approval now includes adult and pediatric patients as young as 2 years old. The decision is an important milestone as it offers early therapeutic intervention for young patients suffering from severe obesity and hyperphagia caused by MC4R pathway disorders. Early treatment is crucial to prevent further health complications associated with obesity. The expanded use of IMCIVREE aligns with broader efforts to address and manage rare genetic conditions affecting energy balance and appetite control, with availability expected through the Medicines for Children program by year’s end.
Read Announcement- Drug:
- IMCIVREE (setmelanotide)
- Announced Date:
- August 26, 2024
- Target Action Date:
- December 26, 2024
- Indication:
- Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency
Announcement
Rhythm Pharmaceuticals, Inc announced that The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 26, 2024. .
AI Summary
Rhythm Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review for its supplemental New Drug Application (sNDA) for IMCIVREE (setmelanotide). This review is focused on the treatment of obesity due to Bardet-Biedl syndrome or POMC/LEPR deficiency in young children as young as 2 years old. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of December 26, 2024, marking an important step toward providing a precision therapy for children suffering from hyperphagia and severe obesity caused by these rare genetic conditions. The decision highlights the potential for earlier treatment, which may improve the quality of life for affected children and their families by addressing abnormal hunger and weight issues at a younger age.
Read Announcement- Drug:
- IMCIVREE (setmelanotide)
- Announced Date:
- August 26, 2024
- Indication:
- Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency
Announcement
Rhythm Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for the treatment of obesity due to Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency obesity in children as young as 2 years old.
AI Summary
Rhythm Pharmaceuticals announced that the FDA has accepted its supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide) to treat obesity linked to Bardet-Biedl syndrome (BBS) or genetic obesity due to POMC, including PCSK1, and LEPR deficiency. The treatment is now proposed for children as young as 2 years old. This sNDA is supported by data from a Phase 3 trial involving 12 patients under 6 years old, which showed encouraging improvements in reducing BMI and managing hyperphagia—a condition marked by uncontrollable hunger and abnormal food-seeking behavior.
The FDA has granted Priority Review for the application and set a Prescription Drug User Fee Act (PDUFA) goal date of December 26, 2024. If approved, this could provide a new, targeted therapy for very young patients, potentially improving their quality of life and offering relief to families managing these rare, genetically driven conditions.
Read Announcement
Bivamelagon FDA Regulatory Events
Bivamelagon is a drug developed by Rhythm Pharmaceuticals for the following indication: In patients with acquired hypothalamic obesity.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- bivamelagon
- Announced Date:
- July 9, 2025
- Indication:
- In patients with acquired hypothalamic obesity.
Announcement
Rhythm Pharmaceuticals, Inc. announced positive topline results from its Phase 2 trial evaluating bivamelagon (formerly LB54640), an investigational oral melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals, Inc. recently announced positive topline results from its Phase 2 trial evaluating bivamelagon (formerly LB54640), an investigational oral melanocortin-4 receptor (MC4R) agonist. The study focused on patients with acquired hypothalamic obesity, a condition linked to damage in the brain’s hunger control center. It was a randomized, placebo-controlled, double-blind trial that enrolled 28 patients aged 12 and older. Participants received either a low (200 mg), middle (400 mg), or high dose (600 mg) of bivamelagon, or a placebo, on a daily basis over a 14-week period. The primary goal was to measure the change in body mass index from the start of the trial. The promising topline data highlights the potential of bivamelagon to improve weight management, decrease hunger, and enhance quality of life for those affected by hypothalamic obesity.
Read Announcement- Drug:
- bivamelagon
- Announced Date:
- July 8, 2025
- Indication:
- In patients with acquired hypothalamic obesity.
Announcement
Rhythm Pharmaceuticals, Inc announced the Company will hold a conference call and webcast on Wednesday, July 9, 2025 at 8:00 a.m. ET to disclose topline results from the randomized, placebo-controlled portion of the Phase 2 trial evaluating bivamelagon, an investigational oral melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals announced that it will hold a conference call and webcast on Wednesday, July 9, 2025 at 8:00 a.m. ET. During the call, the company will share topline results from the randomized, placebo-controlled portion of its Phase 2 trial. This trial is testing bivamelagon, an investigational oral melanocortin-4 receptor (MC4R) agonist, in patients with acquired hypothalamic obesity. The study involves patients who are 12 years and older. Participants in the trial received daily doses of bivamelagon at low (200 mg), middle (400 mg), or high (600 mg) levels, or a placebo, over a 14-week period. The main goal of the research is to assess changes in body mass index from the start of the trial. The live conference call and webcast, which will later be available as an archived version, will provide more details on these clinical findings.
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LB54640 FDA Regulatory Events
LB54640 is a drug developed by Rhythm Pharmaceuticals for the following indication: In hypothalamic obesity.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- LB54640
- Announced Date:
- July 23, 2024
- Indication:
- In hypothalamic obesity.
Announcement
Rhythm Pharmaceuticals, Inc. announced that the first patients have been dosed in the Company's Phase 2 clinical trial evaluating LB54640, an investigational oral melanocortin-4 receptor (MC4R) agonist, in hypothalamic obesity.
AI Summary
Rhythm Pharmaceuticals, Inc. announced that the first patients have been dosed in their Phase 2 clinical trial evaluating LB54640, an investigational oral melanocortin-4 receptor (MC4R) agonist, targeting hypothalamic obesity. The trial is designed as a randomized, placebo-controlled, double-blind study with patients 12 years and older receiving one of three different daily oral doses or a placebo for 14 weeks. The primary goal is to measure changes in body mass index, along with effects on hunger, safety, and quality of life.
Based on encouraging preclinical data and positive Phase 1 results, LB54640 could offer a new oral treatment option for patients struggling with hyperphagia and severe obesity. Rhythm Pharmaceuticals hopes that this study will further demonstrate the potential of LB54640 to treat MC4R pathway diseases effectively, without the side effects, such as hyperpigmentation, seen with some other therapies.
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