Keros Therapeutics, Inc. announced on August 20, 2025, that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (DMD). This designation highlights the significant unmet medical need for patients suffering from this rare disease.
The Orphan Drug designation provides several potential benefits, including tax credits for qualified clinical testing expenses and a waiver or partial payment of FDA application fees. Crucially, if KER-065 is approved, this designation could provide seven years of market exclusivity.
This regulatory milestone is significant for Keros as it advances KER-065 towards a Phase 2 clinical trial in patients with DMD. KER-065 is designed to inhibit myostatin and activin A, aiming to increase skeletal muscle regeneration, muscle size and strength, and improve bone strength.
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