Setmelanotide

also known as RM-493 · BIM-22493 · Imcivree · RM-493 ACETATE · Setmelanotida · SETMELANOTIDE ACETATE

Protein / enzyme biologic targets MC4R via activation

Developed for: Bardet-Biedl syndrome · inherited obesity · obesity disorder · acquired hypothalamic obesity · obesity due to pro-opiomelanocortin deficiency · Alstrom syndrome · obesity due to leptin receptor gene deficiency · Prader-Willi syndrome
+5 more
· hypertriglyceridemia · obesity due to melanocortin 4 receptor deficiency · obesity due to prohormone convertase I deficiency · pseudohypoparathyroidism type 1A · morbid obesity
Investigated by: Rhythm Pharmaceuticals, Inc. · Charite University, Berlin, Germany · Massachusetts General Hospital · Tom Hühne

Regulatory milestones approvals, filings & regulatory actions · 15 recorded

Milestone	Jurisdiction	Brand	Indication	Date	Sentence it was read from
Filed Bardet-Biedl syndrome 1 milestone
Filed	EU (EMA)	IMCIVREE	Bardet-Biedl syndrome	2021-10-14	“withdraw the proposed Alström syndrome indication from its pending Type II variation application to the European Medicines Agency (EMA) for setmelanotide for the treatment of...” rhythmtx.com ↗ “submitted a Type II variation application to the European Medicines Agency in October 2021 seeking regulatory approval and authorization for setmelanotide to treat obesity and...” rhythmtx.com ↗ “Marketing Authorization Application (MAA) for setmelanotide to treat individuals living with POMC deficiency obesity or LEPR deficiency obesity to the European Medicines Agency (EMA)” rhythmtx.com ↗ “The European Medicines Agency (EMA) is reviewing Rhythm’s Type II variation submission to the Marketing Authorization Application (MAA) for setmelanotide for the same indication.” rhythmtx.com ↗ “submission of its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the treatment of POMC deficiency obesity and LEPR deficiency obesity” rhythmtx.com ↗ “and submitted a Type II variation application to the European Medicines Agency in October 2021 seeking regulatory approval and authorization for setmelanotide” rhythmtx.com ↗ “The Company submitted a Type II variation application to the European Medicines Agency seeking regulatory approval and authorization for setmelanotide” rhythmtx.com ↗ “today announced that it has submitted its Type II variation application to the European Medicines Agency (EMA) for IMCIVREE ® (setmelanotide)” rhythmtx.com ↗
Filed obesity disorder 1 milestone
Filed	EU (EMA)	—	obesity disorder	2024-01-04	“submitted a Type II variation application to the EMA seeking regulatory approval and authorization for setmelanotide to treat obesity and control hunger in pediatric patients” rhythmtx.com ↗
Rejected Alstrom syndrome 1 milestone
Rejected (CRL)	US (FDA)	IMCIVREE	Alstrom syndrome	2022-06-16	“Rhythm also announced that the FDA issued a complete response letter for the sNDA for setmelanotide in Alström syndrome.” rhythmtx.com ↗
Approved Indication not stated 10 milestones
Filed	Japan (PMDA)	—	acquired hypothalamic obesity	2026-05-05	“Japanese New Drug Application (JNDA) for setmelanotide to treat acquired HO has been accepted, validated and is now under review” rhythmtx.com ↗
Label expansions (3)	US (FDA)	IMCIVREE	—	2022-06-16 – 2026-03-19	fda.gov ↗
Filed	EU (EMA)	—	acquired hypothalamic obesity	2025-08-20	“the European Medicines Agency (EMA) has confirmed validation of the Type II variation submission to the Marketing Authorization Application (MAA) for setmelanotide” rhythmtx.com ↗
Filed	US (FDA)	—	acquired hypothalamic obesity	2025-08-20	“has accepted for filing the company’s supplemental New Drug Application (sNDA) for setmelanotide seeking approval for the treatment of conditions associated with acquired...” rhythmtx.com ↗
Filed	US (FDA)	IMCIVREE	obesity due to Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiency	2024-08-06	“Completed sNDA submission seeking to expand U.S. label of IMCIVREE to treat pediatric patients as young as 2 years old in approved indications” rhythmtx.com ↗
Filed	Other	—	Bardet-Biedl syndrome, POMC deficiency, PCSK1 deficiency, LEPR deficiency	2022-11-21	“In November 2022 , Rhythm announced that Health Canada accepted for review its New Drug Submission (NDS) with Priority Review for setmelanotide” rhythmtx.com ↗ “Health Canada has accepted for review its New Drug Submission (NDS) for setmelanotide” rhythmtx.com ↗
Filed	US (FDA)	IMCIVREE	Bardet-Biedl syndrome, Alström syndrome	2021-11-15	“FDA has accepted for filing the company’s supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide)” rhythmtx.com ↗
Filed	US (FDA)	IMCIVREE	Bardet-Biedl syndrome, Alström syndrome	2021-09-20	“FDA) has accepted for filing Rhythm’s New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity” rhythmtx.com ↗ “FDA) accepted the company's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity” rhythmtx.com ↗ “completed its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for IMCIVREE ® (setmelanotide)” rhythmtx.com ↗ “March 20, 2026 PDUFA goal date for sNDA for setmelanotide in acquired hypothalamic obesity” rhythmtx.com ↗
Approved	EU (EMA)	Imcivree	—	2021-07-16	europa.eu ↗
Approved	US (FDA)	IMCIVREE	—	2020-11-25	fda.gov ↗

Trials 23

today

US approved 2020

EU approved 2021

NCT07496463 · Ph 2 NCT06760546 · Ph 3 NCT06772597 · Ph 2 NCT06596135 · Ph 3 NCT05774756 · Ph 3 NCT07674290 · Ph 4 NCT04966741 · Ph 3 NCT05194124 · Ph 3 NCT05093634 · Ph 3 NCT04963231 · Ph 2 NCT05046132 · Ph 4 NCT04725240 · Ph 2 NCT04348175 · Ph 1 NCT03746522 · Ph 3 NCT03651765 · Ph 2/3 NCT03287960 · Ph 3 NCT03262610 · Ph 2 NCT02896192 · Ph 3 NCT03013543 · Ph 2 NCT02311673 · Ph 2 NCT02507492 · Ph 2 NCT01749137 · Ph 2

2013201420152016201720182019202020212022202320242025202620272028202920302031

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 11 trial
Phase 1	NCT04348175	Jul 2020 → Apr 2021	impaired renal function disease	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 29 trials · 1 met primary · 2 missed
Phase 2	NCT07496463	Apr → Oct 2026 expected	obesity disorder, pseudohypoparathyroidism type 1A	Massachusetts General Hospital	Not yet recruiting	No outcome recorded
Phase 2	NCT06772597	Mar 2025 → Oct 2026 expected	Prader-Willi syndrome, obesity disorder	Rhythm Pharmaceuticals, Inc.	Active not recruiting	No outcome recorded
Phase 2	NCT04963231	Nov 2021 → Aug 2023	inherited obesity	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 2	NCT04725240	Jun 2021 → Jun 2022	acquired hypothalamic obesity	Rhythm Pharmaceuticals, Inc.	Completed	Met primary
Phase 2	NCT03262610	Aug 2017 → Jan 2018	hypertriglyceridemia, obesity disorder	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 2	NCT03013543	Feb 2017 → Mar 2022	obesity due to melanocortin 4 receptor deficiency	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 2	NCT02311673	Mar 2015 → Oct 2016	Prader-Willi syndrome	Rhythm Pharmaceuticals, Inc.	Completed	Missed primary
Phase 2	NCT02507492	Jan 2015 → Jan 2018	inherited obesity	Charite University, Berlin, Germany	Unknown	No outcome recorded
Phase 2	NCT01749137	Jan → Sep 2013	obesity disorder	Rhythm Pharmaceuticals, Inc.	Completed	Missed primary
Phase 2/31 trial
Phase 2/3	NCT03651765	Jul 2018 → Jan 2025	inherited obesity	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 39 trials · 4 met primary · 1 missed
Phase 3	NCT06760546	Sep 2025 → Mar 2027 expected	acquired hypothalamic obesity, isolated optic nerve hypoplasia, non-acquired combined pituitary hormone deficiency, pituitary stalk interruption syndrome +1	Rhythm Pharmaceuticals, Inc.	Recruiting	No outcome recorded
Phase 3	NCT06596135	Oct 2024 → Jun 2026	inherited obesity	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 3	NCT05774756	Apr 2023 → Mar 2025 overdue	acquired hypothalamic obesity	Rhythm Pharmaceuticals, Inc.	Active not recruiting	Met primary
Phase 3	NCT04966741	Mar 2022 → Sep 2023	Bardet-Biedl syndrome, obesity due to leptin receptor gene deficiency, obesity due to pro-opiomelanocortin deficiency, obesity due to prohormone convertase I deficiency	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 3	NCT05194124	Dec 2021 → Oct 2023	Bardet-Biedl syndrome, obesity due to pro-opiomelanocortin deficiency	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 3	NCT05093634	Dec 2021 → Dec 2025 overdue	inherited obesity	Rhythm Pharmaceuticals, Inc.	Active not recruiting	Missed primary
Phase 3	NCT03746522	Nov 2018 → Nov 2020	Alstrom syndrome, Bardet-Biedl syndrome	Rhythm Pharmaceuticals, Inc.	Completed	Met primary
Phase 3	NCT03287960	Jan 2018 → Sep 2020	obesity due to leptin receptor gene deficiency	Rhythm Pharmaceuticals, Inc.	Completed	Met primary
Phase 3	NCT02896192	Feb 2017 → May 2020	obesity due to pro-opiomelanocortin deficiency	Rhythm Pharmaceuticals, Inc.	Completed	Met primary
Phase 42 trials
Phase 4	NCT07674290	Jan 2023 → Dec 2030 expected	Alstrom syndrome, Bardet-Biedl syndrome	Tom Hühne	Recruiting	No outcome recorded
Phase 4	NCT05046132	Aug 2021 → Apr 2022	—	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase not stated1 trial
—	NCT05183802	—	Bardet-Biedl syndrome, obesity disorder	Rhythm Pharmaceuticals, Inc.	Approved for marketing	No outcome recorded
Comparator or background therapy4 trials
Phase 1	NCT01867437 Comparator	May 2013 → Mar 2014	morbid obesity	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1	NCT02431442 Comparator	Jan 2012 → Jan 2014	obesity disorder	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 1/2	NCT02041195 Comparator	Jan → Aug 2014	morbid obesity	Rhythm Pharmaceuticals, Inc.	Completed	No outcome recorded
Phase 4	NCT05812183 Background	Jul 2023 → Feb 2025	—	University of California, Los Angeles	Withdrawn	No outcome recorded

News releases announcing trial results or a regulatory action · 107

Date	Issuer	Release
2026-09-22	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Health Canada Approval of IMCIVREE® (setmelanotide) for Weight Management in Adult and Pediatric Patients 4 years of Age and Older with Acquired Hypothalamic Obesity rhythmtx.com ↗ 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 Health Canada has approved IMCIVREE ® (setmelanotide) for the additional indication of weight management in adult and pediatric patients 4 years of age and older with acquired hypothalamic obesity (HO).
2026-09-10	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces New Data Presentations at the European Society for Paediatric Endocrinology Meeting rhythmtx.com ↗ The mean change in Lipid Accumulation Product (LAP) (n=41 vs n=22) from baseline was -25.6 compared with +3.6 for the placebo group (p<0.0001).
2026-08-24	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces MHLW Approval of IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity (HO) in Japan rhythmtx.com ↗
2026-08-11	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces IMCIVREE® (setmelanotide) Receives Expanded Marketing Authorization in the United Kingdom for Treatment of Obesity and Control of Hunger in Patients with Acquired Hypothalamic Obesity due to Hypothalamic Injury or Impairment rhythmtx.com ↗ 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 the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) 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 older with acquired hypothalamic obesity (HO) due to hypothalamic injury or impairment.
2026-07-08	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces The New England Journal of Medicine Publication of Phase 3 TRANSCEND Trial Results in Acquired Hypothalamic Obesity rhythmtx.com ↗ The publication highlights robust improvements in weight and hunger achieved with setmelanotide therapy in adult and pediatric patients aged four years and older.
2026-06-15	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Multiple New Data Presentations from MC4R Agonists in Acquired Hypothalamic Obesity (HO), Bardet-Biedl Syndrome (BBS) and Prader-Willi Syndrome (PWS) at ENDO 2026 rhythmtx.com ↗ Setmelanotide therapy achieved robust, sustained and clinically significant weight loss in patients with acquired HO at 2.5 years
2026-06-13	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Positive Interim Six-month Data from Phase 2 Trial of Setmelanotide in Patients with Prader-Willi Syndrome (PWS) at ENDO 2026 rhythmtx.com ↗ Patients with PWS treated with setmelanotide therapy (N=17) achieved clinically meaningful BMI or BMI z-score reductions, reductions in fat mass with preservation of lean mass, and improvements in hyperphagia and anxiety measures
2026-05-12	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces New Data Presentations at the 2026 European Congress of Endocrinology rhythmtx.com ↗ Setmelanotide treatment in patients with acquired HO led to significant improvements in multiple metabolic index scores versus placebo.
2026-05-04	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces New Data Presentations in Acquired Hypothalamic Obesity at Pediatric Endocrine Society rhythmtx.com ↗ Pediatric patients with acquired hypothalamic obesity who received setmelanotide for up to 2.5 years achieved sustained reductions across multiple age‑adjusted weight‑related measures.
2026-05-01	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces IMCIVREE® (Setmelanotide) Granted Marketing Authorization by European Commission for the Treatment of Obesity and Control of Hunger in Patients with Acquired Hypothalamic Obesity rhythmtx.com ↗ European Commission grants authorization in adults and children 4 years of age and above with acquired hypothalamic obesity due to hypothalamic injury or impairment
2026-03-26	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Positive CHMP Opinion for IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Patients with Acquired Hypothalamic Obesity due to Hypothalamic Injury or Impairment rhythmtx.com ↗
2026-03-19	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity rhythmtx.com ↗ 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 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).
2026-03-16	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Topline Results from Phase 3 EMANATE Trial rhythmtx.com ↗ The four substudies of this global, Phase 3 trial evaluating setmelanotide did not meet primary endpoints.
2026-03-01	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Additional Positive Data from Phase 3 TRANSCEND trial of Setmelanotide in Patients with Acquired Hypothalamic Obesity rhythmtx.com ↗ Among patients aged 12 and older (n=98), the setmelanotide group (n=66) showed an average weekly reduction of 2.5 points in the weekly average most hunger score, compared with a 1.3‑point reduction in the placebo group (n=32) (p=0.0015).
2025-12-11	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Preliminary Data from Exploratory Phase 2 Trial that showed Setmelanotide Demonstrated Positive Efficacy Signal in Prader-Willi Syndrome rhythmtx.com ↗ 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 positive preliminary results from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS).
2025-11-10	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Presentation of Four Datasets at ObesityWeek® 2025 rhythmtx.com ↗ Phase 3 TRANSCEND trial data showed setmelanotide achieved significant BMI reductions in patients with acquired hypothalamic obesity on concomitant treatment with GLP-1 therapy
2025-11-07	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Extension of Review Period for IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity rhythmtx.com ↗ today 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.
2025-08-20	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Acceptance of sNDA for Setmelanotide in Acquired Hypothalamic Obesity rhythmtx.com ↗
2025-07-12	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Data on MC4R Agonists Setmelanotide and Bivamelagon at ENDO 2025 rhythmtx.com ↗ Full data from Phase 3 TRANSCEND study underscore potential efficacy of setmelanotide, including with prior use or concomitant use of GLP-1s
2025-05-14	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces New Data Presentations on Patients with Acquired or Congenital Hypothalamic Obesity (N=35) Treated with Setmelanotide for up to Nine Months in French Early-access Program rhythmtx.com ↗ New data showed setmelanotide achieved consistent, meaningful weight reduction
2025-04-07	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Pivotal Phase 3 TRANSCEND Trial Meets Primary Endpoint with -19.8% Placebo-adjusted BMI Reduction in Patients (N=120) with Acquired Hypothalamic Obesity rhythmtx.com ↗ The global trial met its primary endpoint with a statistically significant and highly clinically meaningful reduction in body mass index (BMI) with setmelanotide in both adult and pediatric patients versus placebo.
2025-03-19	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Orphan Drug Designation Granted to Setmelanotide for Treatment of Hypothalamic Obesity in Japan rhythmtx.com ↗ today 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.
2024-12-20	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients as Young as 2 Years Old rhythmtx.com ↗ 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.
2024-12-03	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces ▼ IMCIVREE® (setmelanotide) Receives Expanded Marketing Authorization in the United Kingdom for Treatment of Obesity and Control of Hunger in Patients with Bardet Biedl Syndrome or POMC, PCSK1, or LEPR Deficiency to include Children as Young as 2 Years Old rhythmtx.com ↗ With this decision by the MHRA, patients as young as 2 years old with MC4R pathway diseases can now access a treatment option intended to address the underlying cause of their disease.
2024-11-18	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Five Presentations at ESPE 2024, including New Real-world Data in Pediatric Patients with Acquired or Congenital Hypothalamic Obesity Treated with Setmelanotide rhythmtx.com ↗ today 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.
2024-11-13	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Study of Setmelanotide in Patients Between 2 and 5 Years Old in The Lancet Diabetes & Endocrinology rhythmtx.com ↗ Clinically meaningful improvements were observed in both co-primary endpoints at week 52.
2024-11-04	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Five New Data Presentations at ObesityWeek® 2024 rhythmtx.com ↗ today announced the presentation of new, real-world data showing improvements in hunger scores and body mass index (BMI) reductions in adult patients in France with acquired hypothalamic obesity who were treated with setmelanotide.
2024-08-26	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Acceptance for Priority Review of Supplemental New Drug Application for IMCIVREE® (setmelanotide) in Patients as Young as 2 years old rhythmtx.com ↗ The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 26, 2024 .
2024-08-06	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Reports Second Quarter 2024 Financial Results and Business Update rhythmtx.com ↗ Completed sNDA submission seeking to expand U.S. label of IMCIVREE to treat pediatric patients as young as 2 years old in approved indications
2024-07-31	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces European Commission Expands ▼IMCIVREE® (setmelanotide) Marketing Authorization to include Children as Young as 2 Years Old rhythmtx.com ↗ IMCIVREE is the first-ever authorized precision medicine in the European Union for control of hunger and treatment of obesity in adults and children, now as young as 2 years old, living with BBS or POMC, PCSK1, or LEPR deficiency.
2024-06-28	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Positive CHMP Opinion for ▼IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Patients between 2 and 6 years old with Bardet Biedl Syndrome or POMC, PCSK1, or LEPR Deficiency rhythmtx.com ↗ has adopted a positive opinion recommending the marketing authorization for IMCIVREE ® (setmelanotide) include children between 2 and younger than 6 years old
2024-06-03	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Patient-reported Experiences with Hyperphagia in Hypothalamic Obesity at ENDO 2024 rhythmtx.com ↗ Participants from the completed Phase 2 hypothalamic obesity trial reported positive changes in sleep, energy levels during setmelanotide therapy in qualitative interviews
2024-05-22	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Positive Recommendation from NICE for ▼IMCIVREE® (setmelanotide) for Treatment of Obesity and Hyperphagia in Patients with Bardet-Biedl Syndrome rhythmtx.com ↗
2024-05-06	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Data from Phase 3 Pediatrics Trial at Pediatric Endocrine Society Annual Meeting rhythmtx.com ↗ Treatment with setmelanotide achieved the primary endpoint with a 3.04 mean reduction in BMI-Z score (a measure of body mass index deviations from what is considered normal) and 18.4 percent mean reduction in body mass index (BMI).
2024-04-29	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Publication of Results from Phase 2 Study of Setmelanotide for the Treatment of Hypothalamic Obesity in The Lancet Diabetes & Endocrinology rhythmtx.com ↗ In this first study to investigate the use of setmelanotide as a targeted treatment for hypothalamic obesity, we observed a consistent reduction in body weight and hunger in all adherent patients.
2024-02-07	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Positive Reimbursement Decision in Italy for IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome rhythmtx.com ↗ today announced that the Italian Medicine Agency (AIFA) approved reimbursement ( Gazzetta Ufficiale ) for IMCIVREE ® (setmelanotide) for the treatment of obesity and control of hunger associated with Bardet-Biedl syndrome (BBS).
2023-12-06	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Updates on MC4R Pathway Programs at R&D Event rhythmtx.com ↗ Phase 3 trial evaluating setmelanotide in pediatric patients ages 2-<6yo (N=12) over 52 weeks achieved primary endpoint
2023-11-02	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Positive CADTH Reimbursement Recommendation for IMCIVREE® (setmelanotide) rhythmtx.com ↗
2023-10-17	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Data from its Long-term Extension Study of Setmelanotide Showing Sustained and Deepened BMI Reduction in Patients with Hypothalamic Obesity at One Year at ObesityWeek® 2023 rhythmtx.com ↗ Patients with hypothalamic obesity (n=12) achieved mean BMI reduction of 25.5% at one year on setmelanotide treatment
2023-09-19	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Orphan Drug Designation from European Medicines Agency for Setmelanotide for Treatment of Acquired Hypothalamic Obesity rhythmtx.com ↗ Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced that the European Medicines Agency (EMA) has issued a positive opinion on the Company’s orphan drug designation request for setmelanotide as a treatment for acquired hypothalamic obesity.
2023-08-07	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Pre-Marketing Early Access Authorization for Setmelanotide for Use in Patients with Hypothalamic Obesity in France rhythmtx.com ↗ The AP1 for setmelanotide was granted following review of efficacy and safety data from Rhythm’s Phase 2 trial by the ANSM and HAS.
2023-06-17	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Data Demonstrating BMI Reduction in Patients with Hypothalamic Obesity Treated with Setmelanotide over Six Months at ENDO 2023 rhythmtx.com ↗
2023-05-08	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Health Canada Approval of IMCIVREE® Setmelanotide Injection for Weight Management in Bardet-Biedl Syndrome or Genetically-confirmed Biallelic POMC, PCSK1, or LEPR Deficiency rhythmtx.com ↗
2023-04-24	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Commercial Launch of IMCIVREE® (setmelanotide) in Germany for the Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome rhythmtx.com ↗ The German Federal Joint Committee (G-BA) ruled that IMCIVREE is eligible for reimbursement by Statutory Health Insurances for BBS.
2023-03-27	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Publication of Bardet-Biedl Syndrome Patient and Caregiver Perspectives of Hunger and Quality of Life with Setmelanotide rhythmtx.com ↗ Our analyses also demonstrate that setmelanotide therapy resulted in meaningful improvements for these patients, such as weight loss and a decrease in obsessive focus on food and food-seeking behaviors.
2023-01-23	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Publication of Analysis of Quality of Life Improvements from Phase 3 Clinical Trial in Bardet-Biedl Syndrome rhythmtx.com ↗ Patients reported clinically meaningful improvements across multiple health-related quality of life measures following 52 weeks of treatment with setmelanotide
2022-11-21	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces ▼IMCIVREE® (setmelanotide) Granted Marketing Authorization in Great Britain for Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome rhythmtx.com ↗
2022-11-08	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trial of IMCIVREE® (setmelanotide) in Bardet-Biedl Syndrome in The Lancet Diabetes and Endocrinology rhythmtx.com ↗ The trial met its primary endpoint and all key secondary endpoints, with statistically significant reductions in weight and hunger at 52 weeks on therapy.
2022-11-03	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces 11 Presentations at The Obesity Society’s Annual Meeting at ObesityWeek® rhythmtx.com ↗ 84% of patients with BBS in Phase 3 clinical trial showed clinical benefit of improvement in one measure or more of weight, hunger, or QoL following setmelanotide treatment
2022-11-02	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Data from Phase 2 and Long-term Extension Trials Evaluating Setmelanotide for the Treatment of Hypothalamic Obesity at ObesityWeek® and Plans to Initiate Phase 3 Trial in Early 2023 rhythmtx.com ↗ Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced positive Phase 2 results and long-term extension trial data in hypothalamic obesity that are being presented today as a late-breaking poster at The Obesity Society’s ObesityWeek ® 2022 conference in San Diego .
2022-11-01	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Setmelanotide Granted Breakthrough Therapy Designation for Hypothalamic Obesity by the U.S. FDA rhythmtx.com ↗ setmelanotide received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of hypothalamic obesity.
2022-09-19	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents New Disease Burden Findings and Analyses of Setmelanotide Data in Children and Adolescents with Rare MC4R Pathway Diseases at ESPE 2022 rhythmtx.com ↗ New analyses of Phase 3 data showed setmelanotide achieved substantial weight loss benefit in adolescent and pediatric patients (n=26) with severe obesity due to BBS or POMC or LEPR deficiency
2022-09-06	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces IMCIVREE® (setmelanotide) Granted Marketing Authorization by European Commission for Treatment of Obesity and Control of Hunger in Bardet-Biedl Syndrome rhythmtx.com ↗
2022-07-22	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Positive CHMP Opinion for ▼¹ IMCIVREE® (setmelanotide) for the Treatment of Obesity and Control of Hunger in Patients with Bardet-Biedl Syndrome rhythmtx.com ↗
2022-07-20	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Early Access Authorization for Setmelanotide for Use in Patients with Bardet-Biedl Syndrome in France rhythmtx.com ↗ The AP1 for setmelanotide was granted following review of efficacy and safety data from clinical studies by the ANSM and HAS.
2022-07-18	Rhythm Pharmaceuticals, Inc.	Regulatory NICE Recommends Rhythm’s ▼IMCIVREE® (setmelanotide) for Treating Obesity and Controlling Hunger Caused by POMC or LEPR Deficiency rhythmtx.com ↗ With this recommendation under the Highly Specialised Technologies (HST) pathway, IMCIVREE will be funded and available for use within 90 days in the National Health Service.
2022-07-12	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Positive Interim Results from Phase 2 Clinical Trial Evaluating Setmelanotide in Hypothalamic Obesity rhythmtx.com ↗
2022-06-22	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces that European Commission Authorized Variation for IMCIVREE® (setmelanotide) to Allow for Dosing in Patients with POMC or LEPR deficiency with Renal Impairment rhythmtx.com ↗ 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) this week authorized a variation for IMCIVREE ® (setmelanotide) that allows for dosing in patients with POMC or LEPR deficiency who have mild, moderate or severe renal impairment.
2022-06-16	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Use in Patients with Bardet-Biedl Syndrome rhythmtx.com ↗ 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 U.S. Food and Drug Administration (FDA) has approved the Company’s supplemental New Drug Application (sNDA) for IMCIVREE ® (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for patients with Bardet-Biedl syndrome (BBS).
2022-06-13	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents New Data from Long-term Extension Trial Evaluating Setmelanotide in Bardet-Biedl Syndrome or POMC or LEPR Deficiency Obesity at ENDO 2022 rhythmtx.com ↗ Responses to our precision therapy are shown to be durable, with both adult and pediatric patients experiencing continued reductions in body weight-related measures throughout the long-term extension.
2022-06-13	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents Initial Data from Long-term Extension Trial Evaluating Setmelanotide in Rare Genetic Diseases of Obesity at ENDO 2022 rhythmtx.com ↗ Consistent with our experience in other rare genetic diseases of obesity, these data demonstrate that ongoing treatment with setmelanotide delivers continued improvement in weight-related measures.
2022-05-02	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents New Data from Phase 3 Trial Evaluating Setmelanotide in Patients with Bardet-Biedl Syndrome (BBS) at the Pediatric Endocrine Society (PES) 2022 Virtual Annual Meeting rhythmtx.com ↗ New data show setmelanotide treatment led to clinically meaningful improvements in lipid parameters, without affecting blood pressure or heart rate in patients with BBS
2022-02-24	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Extension of Review Period for IMCIVREE® (setmelanotide) for Patients with Bardet-Biedl Syndrome and Alström Syndrome rhythmtx.com ↗
2022-02-16	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Positive Interim Data from Long-term Extension Study of Setmelanotide in Bardet-Biedl Syndrome (BBS) rhythmtx.com ↗ Patients with BBS achieved deepened and sustained weight loss at 24 months on setmelanotide therapy
2021-11-15	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Acceptance for Filing and Priority Review of Supplemental New Drug Application for IMCIVREE® (setmelanotide) for Patients with Bardet-Biedl Syndrome and Alström Syndrome rhythmtx.com ↗ The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 16, 2022.
2021-11-01	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents First-ever Data Showing Improvements in Health-related Quality of Life for Patients Living with Bardet-Biedl Syndrome at ObesityWeek® 2021 rhythmtx.com ↗ this week presented the first-ever data on the health-related quality of life (HRQOL) and experience of patients with Bardet-Biedl syndrome (BBS) who were treated in its Phase 3 trial of setmelanotide
2021-10-14	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents New Data on Experience of People Living with Rare Genetic Diseases of Obesity and Provides Updates on Uncovering Rare Obesity® Genetic Testing Program rhythmtx.com ↗ New HRQOL data from post-hoc analyses of Phase 3 trials evaluating setmelanotide in patients with POMC or LEPR deficiency obesity that showed setmelanotide treatment led to sustained, clinically meaningful HRQOL improvements in a majority of patients
2021-10-14	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Completes Submission of Type II Variation Application to the European Medicines Agency for IMCIVREE® (setmelanotide) for Bardet-Biedl and Alström Syndromes rhythmtx.com ↗
2021-09-22	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents New Data from Phase 2 and 3 Trials Evaluating Setmelanotide in Multiple Rare Genetic Diseases of Obesity at the 59th Annual ESPE Meeting rhythmtx.com ↗ New subgroup analysis of Phase 3 data show setmelanotide achieved statistically significant weight loss and hunger reduction compared with placebo at 14 weeks in patients with Bardet-Biedl syndrome
2021-09-21	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Marketing Authorisation of ▼IMCIVREE® (setmelanotide) in Great Britain rhythmtx.com ↗ First-ever authorised treatment option in Great Britain ( England , Scotland and Wales ) for these rare genetic diseases of obesity
2021-09-20	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Completes Submission of Supplemental New Drug Application to U.S. Food and Drug Administration for IMCIVREE® (setmelanotide) for the treatment of obesity and control of hunger in Bardet-Biedl and Alström Syndromes rhythmtx.com ↗ Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that it has completed its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for IMCIVREE ® (setmelanotide) for the treatment of obesity and control of hunger in adult and pediatric patients 6 years of age and older with Bardet-Biedl syndrome (BBS) or Alström syndrome.
2021-07-23	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces European Commission Authorization of IMCIVREE® (setmelanotide)▼ for the Treatment of Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency rhythmtx.com ↗ First-ever authorized treatment option in the European Union for these rare genetic diseases of obesity
2021-05-21	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Positive CHMP Opinion for Setmelanotide for Treatment of Obesity and Control of Hunger Associated with POMC, PCSK1 and LEPR Deficiency rhythmtx.com ↗
2021-03-20	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Presents New Data from Phase 2 Basket Study Showing Continued Weight Loss at Up to Nine Months in Patients with HET Obesity on Setmelanotide at ENDO 2021 rhythmtx.com ↗ Responders with HET obesity achieved mean weight loss of greater than 12 percent at nine months on setmelanotide therapy
2021-01-26	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Positive Data with Setmelanotide in Additional MC4R Pathway Deficiency-related Obesities rhythmtx.com ↗ Clinical data from Phase 2 Basket Study including five cohorts totaling 65 patients demonstrated proof-of-concept in HET POMC, PCSK1 or LEPR deficiencies, SRC1 and SH2B1 deficiency obesities
2020-12-22	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Positive Topline Results from Pivotal Phase 3 Clinical Trial Evaluating Setmelanotide in Bardet-Biedl and Alström Syndromes rhythmtx.com ↗ The study met its primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight and hunger scores.
2020-11-27	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE™ (setmelanotide) as First-ever Therapy for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency rhythmtx.com ↗ With this approval, IMCIVREE becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity.
2020-11-04	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces New Clinical Data on Setmelanotide at ObesityWeek® 2020 rhythmtx.com ↗ Additional data from long-term extension study in POMC deficiency obesity showed durable weight loss and reductions in hunger at up to three years on therapy
2020-10-30	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trials of Setmelanotide in The Lancet Diabetes & Endocrinology rhythmtx.com ↗ As previously reported, data from the studies demonstrate that treatment with setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, led to statistically significant and clinically meaningful reductions of weight and hunger.
2020-07-16	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Publication of Results from Phase 2 Study of Setmelanotide in Bardet-Biedl Syndrome in Diabetes, Obesity and Metabolism rhythmtx.com ↗ As previously reported, data from the study demonstrate that treatment with setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, reduced body weight and hunger in individuals with BBS.
2020-07-01	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration for Setmelanotide for Treatment of POMC and LEPR Deficiency Obesities rhythmtx.com ↗ today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designations for setmelanotide, an investigational melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.
2020-06-24	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Positive Results from Phase 2 Study of Once-weekly Formulation of Setmelanotide in Healthy Obese Volunteers rhythmtx.com ↗ Healthy obese people treated with the weekly formulation of setmelanotide achieved comparable weight loss to those treated with the daily formulation, and both weekly and daily formulations of setmelanotide were observed to be safe and well-tolerated.
2020-05-13	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces FDA Acceptance of New Drug Application for Setmelanotide for the Treatment of POMC and LEPR Deficiency Obesities rhythmtx.com ↗ The FDA granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020 .
2020-03-30	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Completes Rolling Submission of New Drug Application to U.S. Food and Drug Administration for Setmelanotide in POMC and LEPR Deficiency Obesities rhythmtx.com ↗ today announced that it has completed its rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for setmelanotide
2020-03-18	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives Orphan Drug Designation from U.S. FDA for Setmelanotide for the Treatment of Alström Syndrome rhythmtx.com ↗ Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to setmelanotide for the treatment Alström syndrome.
2019-11-04	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Late-breaking Data from Phase 3 Trials of Setmelanotide in POMC and LEPR Deficiency Obesities at ObesityWeek 2019 rhythmtx.com ↗ Data show treatment effect of setmelanotide on additional secondary endpoints, including body mass index
2019-09-25	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Provides Update on Research and Development Programs rhythmtx.com ↗ Updated data from continuing patients with Bardet-Biedl syndrome in Phase 2 Basket trial demonstrate 22.2 percent mean weight loss following approximately two years on therapy
2019-08-07	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Positive Topline Results from Pivotal Phase 3 Clinical Trials Evaluating Setmelanotide in POMC and LEPR Deficiency Obesities rhythmtx.com ↗ Both studies met their primary endpoints and all key secondary endpoints, demonstrating a statistically significant and clinically meaningful effect on weight loss and reductions in insatiable hunger, or hyperphagia, in patients with POMC and LEPR deficiency obesities.
2019-07-29	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Positive Opinion by the European Medicines Agency on Orphan Drug Designation for Setmelanotide for the Treatment of Bardet-Biedl Syndrome rhythmtx.com ↗ the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending setmelanotide for designation as an orphan medicinal product for the treatment of patients with Bardet-Biedl syndrome (BBS).
2019-03-28	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Promising Clinical Data in MC4R Pathway Heterozygous Obesity and Strategy for Further Development rhythmtx.com ↗ Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of rare genetic disorders of obesity, today announced updated interim data from its ongoing Phase 2 basket study of setmelanotide in melanocortin-4 receptor (MC4R) pathway heterozygous (HET) obesity.
2019-01-04	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals to Outline Expected 2019 Milestones and Review Updated Clinical Data in Bardet-Biedl Syndrome at 37th Annual J.P. Morgan Healthcare Conference rhythmtx.com ↗ Updated clinical data from Phase 2 basket studies evaluating setmelanotide in Bardet-Biedl Syndrome (BBS) show continued weight loss for additional two patients at longer-term follow-up
2018-11-15	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Presentation of Updated Clinical Data from Phase 2 Basket Studies Evaluating Setmelanotide in Alström Syndrome at ObesityWeek 2018 rhythmtx.com ↗ As previously reported, these results continue to show that treatment with setmelanotide reduced body weight and decreased appetite in multiple patients with Alström Syndrome.
2018-09-28	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Presentation of Updated Clinical Data from Phase 2 Basket Studies Evaluating Setmelanotide in Bardet-Biedl Syndrome and Alström Syndrome at 57th Annual ESPE Meeting rhythmtx.com ↗ Results from the studies demonstrate that treatment with setmelanotide led to marked reductions in body weight and decreased appetite as shown by lower hunger scores.
2018-07-23	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Receives European Medicines Agency PRIME Designation for Setmelanotide in Rare Genetic Disorders of Obesity rhythmtx.com ↗
2018-06-18	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces New Topline Clinical Data from Ongoing Phase 2 Basket Studies Evaluating Setmelanotide in Rare Genetic Disorders of Obesity rhythmtx.com ↗ Across all these rare genetic disorders of obesity, setmelanotide was observed to be well-tolerated.
2018-05-14	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Reports First Quarter 2018 Financial Results rhythmtx.com ↗ U.S. Food and Drug Administration ( FDA ) Agreed that Bardet-Biedl Syndrome (BBS) and Alström Syndrome are Included Under Existing Breakthrough Therapy Designation (BTD) for Setmelanotide
2018-05-09	Rhythm Pharmaceuticals, Inc.	Results Rhythm Pharmaceuticals Announces Nature Medicine Publication of Longer-Term Data from Phase 2 Study of Setmelanotide for Treatment of LEPR Deficiency Obesity rhythmtx.com ↗ The longer-term data from Rhythm’s open label study show that once daily subcutaneous injection of setmelanotide resulted in reductions in hyperphagia and body-weight in three patients with LEPR deficiency obesity.
2018-01-04	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Pharmaceuticals Announces Progress in Clinical Development of Setmelanotide for Treatment of Rare Genetic Disorders of Obesity rhythmtx.com ↗ Setmelanotide granted Orphan Drug Designation for treatment of leptin receptor (LEPR) deficiency obesity
2017-10-31	Rhythm Pharmaceuticals, Inc.	Results Rhythm Announces Preliminary Data from Phase 2 Study of Setmelanotide for Treatment of Bardet-Biedl Syndrome rhythmtx.com ↗ Substantial Weight Loss Observed in 4 of 5 Patients
2017-06-27	Camurus AB	Results Rhythm and Camurus Announce Positive Initial Data for Extended-Release Delivery of Setmelanotide for Treatment of Rare Genetic Disorders of Obesity camurus.com ↗ “The initial results from this first trial are impressive and meet our pharmacokinetics and tolerability criteria for a once-weekly formulation of setmelanotide,” said Dr. Keith Gottesdiener, Chief Executive Officer and Chairman of the Board of Directors of Rhythm.
2017-06-27	Rhythm Pharmaceuticals, Inc.	Results Rhythm and Camurus Announce Positive Initial Data for Extended-Release Delivery of Setmelanotide for the Treatment of Rare Genetic Disorders of Obesity rhythmtx.com ↗ Rhythm and Camurus today announced positive initial results from an ongoing Phase 1A clinical trial evaluating the pharmacokinetics and tolerability of an extended-release formulation of setmelanotide (RM-493), Rhythm's novel melanocortin-4 receptor (MC4R) agonist in development for the treatment of rare genetic disorders of obesity.
2017-05-11	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Receives Expanded FDA Breakthrough Therapy Designation for Setmelanotide for Rare Genetic Disorders of Obesity rhythmtx.com ↗ Rhythm today announced that the U.S. Food and Drug Administration (FDA) has expanded a previously granted Breakthrough Therapy Designation (BTD) for setmelanotide, the company's novel melanocortin-4 receptor (MC4R) agonist.
2016-11-04	Rhythm Pharmaceuticals, Inc.	Results Rhythm Presents Positive Initial Data for Setmelanotide in LepR Deficiency Obesity rhythmtx.com ↗ Rhythm, a biopharmaceutical company developing peptide therapeutics for rare genetic deficiencies that result in life-threatening metabolic disorders, today announced the presentation of results from an ongoing Phase 2 clinical trial assessing the safety and efficacy of setmelanotide (RM-493), the company's novel melanocortin-4 receptor (MC4R) agonist, for the treatment of leptin receptor (LepR) deficiency obesity.
2016-07-20	Rhythm Pharmaceuticals, Inc.	Results Rhythm Announces New England Journal of Medicine Publication of Setmelanotide Phase 2 Data for Treatment of POMC Deficiency Obesity rhythmtx.com ↗ The NEJM publication reports initial data for two patients in the Phase 2 non-randomized, open label clinical trial of setmelanotide for the treatment of POMC deficiency obesity, an ultra-orphan disease that we believe affects an estimated 100-500 people worldwide.
2016-01-07	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Receives Orphan Drug Designation for Setmelanotide for the Treatment of Prader-Willi Syndrome rhythmtx.com ↗ Rhythm announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for setmelanotide (RM-493), the company's novel melanocortin-4 receptor (MC4R) agonist, for the treatment of Prader-Willi syndrome (PWS).
2016-01-07	Rhythm Pharmaceuticals, Inc.	Regulatory Rhythm Receives FDA Breakthrough Therapy Designation for Setmelanotide for Treatment of POMC Deficiency Obesity rhythmtx.com ↗ the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to setmelanotide, the company's novel melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity.
2014-06-23	Rhythm Pharmaceuticals, Inc.	Results Rhythm Presents Clinical Data that RM-493 Increases Energy Expenditure in Obesity Study rhythmtx.com ↗ Analysis of the data indicates that RM-493 increased resting energy expenditure significantly in this trial.

All press releases naming this drug 133 releases

Date	Issuer	Release
Show all 133 releases opens the full record — a long page

Evidence & citations 12 cited values

Every value below carries the sentence it was read from. 29 sources stand behind the page.

Field	Value	Cited text
Known as	Setmelanotide	“Setmelanotide (Imcivree)” NCT05812183 ↗ 23 NCT06596135 ↗ NCT01749137 ↗ NCT04348175 ↗ NCT03287960 ↗ NCT05046132 ↗ NCT07674290 ↗ NCT03651765 ↗ NCT05774756 ↗ NCT04963231 ↗ NCT06772597 ↗ Show 13 more sources
Known as	BIM-22493	ChEMBL registry synonym — accepted as the source's own label CHEMBL3301624 ↗
Known as	Imcivree	“Setmelanotide (Imcivree)” NCT05812183 ↗
Known as	RM-493	ClinicalTrials.gov intervention name — accepted as the source's own label NCT02431442 ↗ 2 NCT02507492 ↗ NCT01867437 ↗
Known as	RM-493 ACETATE	ChEMBL registry synonym — accepted as the source's own label CHEMBL3301624 ↗
Known as	Setmelanotida	ChEMBL registry synonym — accepted as the source's own label CHEMBL3301624 ↗
Known as	SETMELANOTIDE ACETATE	ChEMBL registry synonym — accepted as the source's own label CHEMBL3301624 ↗
Action	Activate	“a Melanocortin 4 Receptor (MC4R) Agonist” NCT02311673 ↗
Modality	Peptide	“RM-493 is a potent, selective peptide agonist for the MC4R” NCT01867437 ↗
Modality	Protein / enzyme biologic	— CHEMBL3301624 ↗
Route	Subcutaneous	“RM-493 (1 mg/24 h) or placebo by continuous subcutaneous infusion over 72 hours” PMID 25675384 ↗ Feb 2015
Target	MC4R	“RM-493 is a potent, selective peptide agonist for the MC4R” NCT01867437 ↗