Prader-Willi syndrome is a complex genetic disorder caused by loss of paternally expressed genes on chromosome 15q11-q13, characterized by hypotonia, hyperphagia, obesity risk, endocrine dysfunction, and developmental challenges.
Rhythm reported Q1 2026 IMCIVREE revenue of $60.1 million and said more than 150 patient start forms were received after U.S. approval in acquired hypothalamic obesity. The company also cited European authorization and a Japanese application under review.
Aardvark Therapeutics has voluntarily paused its Phase 3 HERO trial testing ARD-101 for hyperphagia in Prader-Willi Syndrome patients after reversible cardiac observations were identified during safety monitoring, delaying expected topline data beyond Q3 2026.
Three GLP-1 weight-loss drugs—semaglutide, tirzepatide and retatrutide—reduced weight and improved metabolic health in mice lacking the MC4R gene, offering potential treatment for rare genetic obesity.
Amgen, Celltrion, and Palatin Technologies are advancing obesity drug candidates through clinical development, with trials planned for 2026-2028 targeting liver fat reduction, multiple biological pathways, and rare genetic obesity conditions.
Soleno Therapeutics provided updates on its VYKAT XR commercial launch for Prader-Willi syndrome, reporting reimbursement progress across nearly 48 Medicaid programs and over 600 prescribers, while European regulatory review continues for approximately 9,500 additional patients.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07450664 |
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome |
ENROLLING_BY_INVITATION | |
| NCT07348601 |
A Study of CSTI-500 in Patients With Prader-Willi Syndrome |
RECRUITING | PHASE2 |
| NCT07266324 |
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome. |
NOT_YET_RECRUITING | PHASE2 |
| NCT07219485 |
A Study of Pitolisant in Participants With Prader-Willi Syndrome |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT07197034 |
The Hunger Elimination or Reduction Objective (HERO ) Open -Label Extension (OLE) Trial |
SUSPENDED | PHASE3 |
| NCT07006207 |
Brain Olfactory Pathways in Prader-Willi Syndrome |
NOT_YET_RECRUITING | NA |
| NCT06901245 |
Tirzepatide in PWS, HO and GNSO |
RECRUITING | PHASE4 |
| NCT06877715 |
Autistic Symptomatology and Sensory Profile in Children With Prader-Willi Syndrome |
RECRUITING | |
| NCT06851351 |
Respiratory Restriction in Prader-Willi Syndrome |
COMPLETED | |
| NCT06828861 |
ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial |
SUSPENDED | PHASE3 |
