Also known as: BBS
Bardet-Biedl syndrome is a multisystem autosomal recessive ciliopathy characterized by retinal dystrophy, obesity, postaxial polydactyly, cognitive impairment, hypogonadism/genitourinary abnormalities, and renal disease. Clinical severity and manifestations vary across affected individuals.
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.
Rhythm Pharmaceuticals announced expanded Phase 3 TRANSCEND trial data showing an 18.8% placebo-adjusted BMI reduction at 52 weeks for setmelanotide in acquired hypothalamic obesity, with FDA PDUFA goal date set for March 20, 2026.
The FDA is set to announce decisions in March 2026 on several drug applications, including label expansions for approved therapies and new drug candidates, with some making repeat approval attempts.
The FDA is set to make approval decisions on five drug applications in March 2026, including treatments for psoriatic arthritis, dry eye disease, hypothalamic obesity, cholestatic pruritus, and a rare genetic disorder.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06239064 |
Early Genetic Identification of Obesity |
ACTIVE_NOT_RECRUITING | |
| NCT05400278 |
Characterizing the Genotype and Phenotype in Adults With Bardet-Biedl Syndrome |
COMPLETED | |
| NCT05194124 |
Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway |
COMPLETED | PHASE3 |
| NCT04966741 |
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity |
COMPLETED | PHASE3 |
| NCT04874909 |
Classification, Functional Stratification and Biomarkers in Ciliopathy (CILLICORIRCM) |
COMPLETED | NA |
| NCT04461444 |
COhort for Bardet-Bield Syndrome and Alström Syndrome for Translational Research Monocentric Interventional Study |
RECRUITING | NA |
| NCT03490019 |
Treatment of Bardet-Biedl-Syndrome With Metformin for Evaluation of a Possible Visual Improvement |
WITHDRAWN | PHASE2 |
| NCT02435940 |
Inherited Retinal Degenerative Disease Registry |
RECRUITING | |
| NCT02329210 |
Clinical Registry Investigating Bardet-Biedl Syndrome |
RECRUITING | |
| NCT00213811 |
Bardet-Biedl Syndrome Study: Clinical and Genetic Epidemiology Study in Adults |
COMPLETED |
