Rhythm Pharmaceuticals
Rhythm Pharmaceuticals works on 3 rare diseases tracked on Trial Friend, including Addison Disease, Prader-Willi Syndrome, Stargardt Disease, with 6 recruiting clinical trials and 1 FDA-approved rare disease drug.
Rhythm Pharmaceuticals develops precision medicines targeting the MC4R pathway for rare genetic and hypothalamic obesity. IMCIVREE (setmelanotide) is FDA-approved for obesity caused by POMC, PCSK1, or LEPR deficiency, for Bardet-Biedl syndrome, and for acquired hypothalamic obesity (March 2026 expansion). Prader-Willi syndrome is in Phase 2 clinical development.
Focus areas at Rhythm Pharmaceuticals
As a rare disease specialist, Rhythm Pharmaceuticals has active clinical trial programs and drug development efforts across 3 rare diseases, including Addison Disease, Prader-Willi Syndrome, Stargardt Disease. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Rhythm Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Rhythm Pharmaceuticals is headquartered in Boston, United States, founded in 2008, publicly traded under the ticker symbol RYTM. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Rhythm Pharmaceuticals Drug Pipeline
Rhythm Pharmaceuticals has 6 active clinical trials across 4 development stages, with 6 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Rhythm Pharmaceuticals's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Rhythm Pharmaceuticals Clinical Trials (6)
Active and recruiting clinical trials sponsored by Rhythm Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
Rhythm Pharmaceuticals FDA-Approved Drugs (1)
Medications developed or marketed by Rhythm Pharmaceuticals that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| SETMELANOTIDE Melanocortin 4 Receptor Agonist [EPC] | Imcivree subcutaneous | Nov 25, 2020 |
Rhythm Pharmaceuticals Trial Locations
Rhythm Pharmaceuticals clinical trials are running at 85 sites in 6 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (3)
Diseases targeted by Rhythm Pharmaceuticals's clinical trial and drug development programs
Addison Disease is a rare endocrine disorder in which the adrenal glands fail to produce adequate cortisol and aldosterone due to autoimmune destruction, infection, or other causes. Without treatment,...
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 fe...
Stargardt disease is the most common inherited macular dystrophy, caused primarily by mutations in the ABCA4 gene that lead to toxic accumulation of lipofuscin (a waste byproduct of the visual cycle) ...
Patient Resources
Organizations and resources related to Rhythm Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Rhythm Pharmaceuticals
Common questions about Rhythm Pharmaceuticals's rare disease programs, clinical trials, and treatments.