Rare disease drugmaker · based in Boston, United States · founded 2008 · ticker RYTM · rhythmtx.com
Rhythm Pharmaceuticals works on Prader-Willi Syndrome, one of the rare diseases we track. It has 6 active clinical trials, and 2 are looking for volunteers now. It also has 1 FDA-approved rare disease drug.
Rhythm Pharmaceuticals develops medicines that act on the MC4R pathway, the brain's hunger and weight signal, for rare genetic and hypothalamic obesity. Its drug Imcivree (setmelanotide) is FDA approved for people 2 years and older with Bardet-Biedl syndrome or with POMC, PCSK1 or LEPR deficiency confirmed by a genetic test, and since March 19, 2026 for people 4 years and older with acquired hypothalamic obesity.
Rhythm Pharmaceuticals pipeline
Each row lists the diseases Rhythm Pharmaceuticals is studying at that stage, from the ones closest to approval down to the earliest work.
Approved
Drugs the FDA has approved.
Imcivree (setmelanotide)
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
Hypothalamic Obesity 2 trials recruiting
Phase 2
Mid-size studies that check whether a drug works and at what dose.
Studies that follow people already taking an approved drug.
Bardet Biedl Syndrome (BBS) recruiting
Other studies
Studies with no phase listed, such as registries and natural history studies.
Obesity, Childhood
Rhythm Pharmaceuticals clinical trials
These are Rhythm Pharmaceuticals trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
2 trials looking for volunteers now
Looking for volunteersPhase 4Updated a few months agoNCT07674290
Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity
TestsSetmelanotide
For Bardet Biedl Syndrome (BBS), Bardet Biedl Syndrome, Bardet-Biedl Syndrome (BBS) and Alstrom Syndrome
Bardet-Biedl syndrome (BBS) and other rare disorders associated with impairment of the melanocortin-4 receptor (MC4R) pathway are characterized by severe early-onset obesity, hyperphagia, and substantial morbidity. Setmelanotide, an MC4R agonist, is approved in Europe for selected genetic obesity disorders and reimbursed in Germany for eligible patients. This study aims to evaluate the effectiveness, safety, treatment persistence, metabolic outcomes, and patient-reported outcomes of Setmelanotide under real-world conditions. The registry is designed to allow future inclusion of additional MC4R agonists as they become approved and clinically available. The study will primarily be conducted at University Hospital Essen and will collect longitudinal routine clinical data from pediatric and adult patients receiving MC4R agonist therapy according to approved indications.
Looking for volunteersPhase 3No updates in a whileNCT06760546
A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
TestsSetmelanotide, Placebo
For Hypothalamic Obesity, Multiple Pituitary Hormone Deficiency Genetic Form, Septo-Optic Dysplasia, Optic Nerve Hypoplasia and Childhood-onset Combined Pituitary Hormone Deficiency
This is a sub-study of Study RM-493-040 (NCT05774756).
The goal of this sub-study is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with congenital Hypothalamic Obesity (cHO). To determine how well setmelanotide works and how safe it is, patients with cHO will take a daily injection of either setmelanotide or placebo and complete trial assessments for up to 26 weeks on a therapeutic regimen.
This is a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age. Up to 20 patients are planned to be enrolled. Patients will take a daily subcutaneous dose of open-labelsetmelanotide for up to 52 weeks.
Underway, not taking new volunteersPhase 1/2Recently updatedNCT06239116
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
TestsPart A: RM-718 or placebo (matched to specific RM-718 dose cohort), Part B: RM-718 or placebo (matched to specific RM-718 dose cohort), Part C: RM-718, Part D: RM-718
The purpose of this study is to evaluate the safety, tolerability, and PK of RM-718 in healthy subjects with obesity and in patients with MC4R Pathway Impairment
Underway, not taking new volunteersPhase 3Updated a few months agoNCT05774756
A Trial of Setmelanotide in Acquired Hypothalamic Obesity
TestsSetmelanotide, Placebo
For Hypothalamic Obesity
The goal of this trial is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with acquired Hypothalamic Obesity (HO). To determine how well setmelanotide works and how safe it is, patients with HO will take a daily injection of either setmelanotide or placebo and complete trial assessments for 52 weeks on a therapeutic regimen.
A separate sub-study in patients with congenital HO is detailed under NCT06760546.
Underway, not taking new volunteersHasn't posted an update in over a yearNCT06239064
Early Genetic Identification of Obesity
TestsGenetic testing via blood collection
For Obesity, Childhood, Hyperphagia, Retinopathy, Syndactyly and Polydactyly
TITLE: Whole genetic approach in Early Genetic Identification of Obesity (WEGIO)
DESIGN: Multicenter epidemiological study
STUDY POPULATION: Participants at risk for a syndromic or a monogenic genetic obesity, incl. participants clinically diagnosed with Bardet-Biedl-Syndrome (BBS)
NUMBER OF PARTICIPANTS: 1000 for initial genetic sequencing and app. 40 for the follow-up documentation
COORDINATING INVESTIGATOR: Prof. Dr. Arndt Rolfs
Follow a condition Rhythm Pharmaceuticals is studying
Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Rhythm Pharmaceuticals. Unsubscribe anytime.
You'll get, for Prader-Willi Syndrome:
Trial Alerts, when a new trial opens or a trial changes
Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
Turn either off anytime from the link in every email.
From the openFDA database. Tap a drug for side effects, dosing and help paying.
Imcivree setmelanotide
Approved Nov 25, 2020. Taken by subcutaneous route.
More about Rhythm Pharmaceuticals
A Phase 3 study of setmelanotide in congenital hypothalamic obesity is recruiting (NCT06760546). A Phase 2 study in Prader-Willi syndrome has finished enrolling and continues (NCT06772597). Rhythm is also testing 2 newer MC4R drugs: a bivamelagon study open by invitation only (NCT07156578), and an RM-718 study that has finished enrolling (NCT06239116).
Where Rhythm Pharmaceuticals trials take place
The trials above run at 85 study sites in 6 countries. Tap a country to see its states, cities and hospitals.
Germany
42▼
United States
32▼
United Kingdom
6▼
Japan
3▼
Canada
1▼
Netherlands
1▼
Rare diseases Rhythm Pharmaceuticals works on
Each disease page lists every open trial, not only Rhythm Pharmaceuticals's, plus approved treatments.
Prader-Willi Syndrome is a rare genetic disorder affecting chromosome 15 that results in weak muscle tone, low hormone levels, incomplete sexual development, intellectual disability, and a constant...
How common: Approximately 1 in 10,000 to 30,000 live births
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Rhythm Pharmaceuticals's own site is rhythmtx.com, and our help paying for treatment page lists drugmaker programs and charity funds.
Rhythm Pharmaceuticals develops medicines that act on the MC4R pathway, the brain's hunger and weight signal, for rare genetic and hypothalamic obesity. Its drug Imcivree (setmelanotide) is FDA approved for people 2 years and older with Bardet-Biedl syndrome or with POMC, PCSK1 or LEPR deficiency confirmed by a genetic test, and since March 19, 2026 for people 4 years and older with acquired hypothalamic obesity. A Phase 3 study of setmelanotide in congenital hypothalamic obesity is recruiting (NCT06760546). A Phase 2 study in Prader-Willi syndrome has finished enrolling and continues (NCT06772597). Rhythm is also testing 2 newer MC4R drugs: a bivamelagon study open by invitation only (NCT07156578), and an RM-718 study that has finished enrolling (NCT06239116).
Rhythm Pharmaceuticals has active programs across 1 rare disease: Prader-Willi Syndrome. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Rhythm Pharmaceuticals is a rare disease specialist headquartered in Boston, United States, founded in 2008. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Prader-Willi Syndrome.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Rhythm Pharmaceuticals, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
None that we track as of November 2026. No Rhythm Pharmaceuticals label in the FDA's openFDA database matches a rare disease on Trial Friend, which usually means the company has no approved product yet or its approved drugs are for common conditions.
In development: the trials list on this page shows what Rhythm Pharmaceuticals is recruiting for now, and the FDA calendar lists any application once a decision date is public.
Rhythm Pharmaceuticals was founded in 2008 and is headquartered in Boston, United States. The company has been active for 18 years. It trades publicly under the ticker symbol RYTM. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.