Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to treat hyperphagia and obesity. Rhythm is evaluating setmelanotide in a global Phase 3 trial in patients with acquired hypothalamic ...

The FDA granted orphan drug designation to HLX22 for gastric cancer, advancing global phase 3 trials to improve first-line ...

The FDA has granted approval to an act of compassionate use treatment with the anti-cancer drug Namodenoson for a patient ...

Rhythm Pharmaceuticals received an orphan-drug designation from Japan's Ministry of Health, Labour and Welfare for its treatment for a certain type of obesity.

Evaluate, the leading provider of market insights for the pharmaceutical industry, today released its 2025 Orphan Drug Report highlighting forecasts for the orphan drug market out to 2030. The 2025 ...

Korro Bio (NASDAQ:KRRO) said on Friday that it received U.S. FDA's orphan drug designation for investigational medicine ...

Rhythm Pharmaceuticals (RYTM) announced it has received orphan drug designation from Japan’s Ministry of Health, Labour and Welfare for ...

The FDA has granted orphan drug designation to PEP-010 for pancreatic cancer, advancing its development as a potential novel ...

Bexobrutideg, a first-in-class Bruton’s tyrosine kinase degrader, has been granted orphan drug designation from the FDA in ...

has granted orphan drug designation (ODD) to exidavnemab for the treatment of Multiple System Atrophy (MSA), providing incentives for the development of treatments for rare diseases with a high ...

GRIN Therapeutics, Inc., a leader in the development of therapies to treat serious neurodevelopmental disorders, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug ...