Keros Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to KER-065 for the treatment of Duchenne muscular dystrophy (DMD), a rare and progressive genetic disorder that causes severe muscle degeneration.
KER-065 is being developed as a potential therapy to address the underlying muscle loss associated with DMD, and the company plans to advance the candidate into a Phase 2 clinical trial.
“Receiving Orphan Drug designation for KER-065 highlights the significant unmet medical need for patients with DMD,” said Jasbir S. Seehra, President and CEO of Keros Therapeutics. “This designation serves as a significant milestone for Keros as we move forward in clinical development.”
The FDA’s Orphan Drug designation is reserved for therapies intended to treat rare diseases affecting fewer than 200,000 people in the U.S. It provides developers with a range of incentives, including tax credits for clinical testing, waiver of FDA application fees, and seven years of marketing exclusivity if the drug is approved.
KER-065 targets pathways within the transforming growth factor-beta (TGF-β) family of proteins—central to Keros’s broader pipeline of therapeutics aimed at treating diseases involving dysfunctional signaling in muscle and blood development.