– Murray Stewart, MD, Chief Medical Officer
Disorder | Early-Stage Development | Phase 2 | Phase 3 | Registration U.S./EU | PHASE |
---|---|---|---|---|---|
Setmelanotide Pivotal Studies
Obesity due to POMC/PCSK1 deficiency |
|
REGISTRATION U.S./EU | |||
Obesity due to LEPR deficiency |
|
REGISTRATION U.S./EU | |||
Bardet-Biedl syndrome |
|
PHASE 3 | |||
Alström syndrome |
|
PHASE 3 | |||
Setmelanotide Basket Study
Obesity due to POMC, PCSK1 or LEPR heterozygous deficiency |
|
PHASE 2 | |||
SRC1 deficiency obesity |
|
PHASE 2 | |||
SH2B1 deficiency obesity |
|
PHASE 2 | |||
MC4R deficiency obesity |
|
PHASE 2 | |||
Smith-Magenis syndrome |
|
PHASE 2 | |||
Additional disorders* |
|
PHASE 2 | |||
RM-853
Preclinical |
|
Early Stage |
The FDA has granted Breakthrough Therapy Designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway, which includes Bardet-Biedl syndrome (BBS), and Alström syndrome. Also, the FDA has granted Orphan Drug Designation for setmelanotide for the treatment of obesity due to POMC or LEPR deficiency, BBS and Alström syndrome. The European Medicines Agency (EMA) has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. The European Commission has adopted the EMA’s Committee for Orphan Medicinal Products’ positive opinion and designated setmelanotide as an orphan medicinal product for the treatment of patients with obesity due to POMC or LEPR deficiency, BBS and Alström syndrome.
Rhythm’s Marketing Authorization Application for setmelanotide to treat people living with obesity due to POMC, PCSK1 or LEPR deficiency is currently under review by the EMA.
For more information about Rhythm clinical trials, please visit clinicaltrials.gov
For information about access to investigational therapeutics outside of clinical trials, please visit Policy on Expanded Access to Investigational Drugs
* Rhythm is currently assessing setmelanotide in additional disorders, including POMC epigenetic diseases, LEP and CPE deficiency obesity, as part of investigator-initiated protocols within the basket study. Given the recent discovery of these rare disorders of the MC4R pathway, there is currently limited or no genetic sequencing or epidemiology data that defines prevalence. However, Rhythm believes that these are rare disorders which may be setmelanotide-responsive.
Rhythm Pharmaceuticals, Inc.
222 Berkeley Street, 12th Floor
Boston, MA 02116
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