FDA Grants Orphan Drug Designation to Keros Therapeutics’ KER-065 for Duchenne Muscular Dystrophy Treatment

Keros Therapeutics has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its investigational therapy, KER-065, aimed at treating Duchenne muscular dystrophy (DMD). The announcement highlights a significant regulatory milestone for the company as it advances the development of this potential treatment for the rare genetic disorder.

The FDA’s orphan drug designation is reserved for therapies targeting conditions affecting fewer than 200,000 people in the United States. It provides certain benefits to developers, including tax credits for clinical trial costs, exemption from FDA application fees, and seven years of market exclusivity upon approval. Duchenne muscular dystrophy is a progressive neuromuscular disease caused by mutations in the dystrophin gene, leading to severe muscle weakness and degeneration. KER-065 is currently under investigation as a therapeutic option to address this unmet medical need.

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Source: GO-AI-ne1

Date: August 21, 2025

Author

Mark Chiang