Keros Receives U.S. FDA Orphan Drug Designation for KER-065 in Duchenne Muscular Dystrophy
Thursday, August 21, 2025
Keros Therapeutics has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for its investigational therapy KER-065, developed for the treatment of Duchenne muscular dystrophy (DMD).
KER-065 is a novel ligand trap designed to block the effects of myostatin and activin A, proteins that contribute to muscle breakdown. By doing so, it aims to promote muscle regeneration, improve strength, reduce fibrosis, lower body fat and enhance bone health. The company is advancing the candidate into a Phase 2 clinical trial in patients with DMD.
DMD is the most common form of muscular dystrophy, caused by mutations in the gene responsible for producing dystrophin, a protein that supports muscle stability. The absence of dystrophin leads to progressive muscle degeneration, loss of mobility, respiratory problems and cardiomyopathy, which often results in heart failure. The condition mainly affects boys, with around one in every 3,500 male births worldwide impacted.
The designation recognises the urgent need for effective treatments for DMD, a rare and severe muscle-wasting condition. It also offers incentives such as tax credits for clinical trials, waiver or reduction of FDA application fees, and seven years of market exclusivity if the therapy is approved.
Source: kerostx.com
