Keros Therapeutics' Duchenne Muscular Dystrophy Treatment Gets FDA's Orphan Drug Designation

(RTTNews) - Keros Therapeutics, Inc. (KROS), a clinical-stage biopharmaceutical company, Wednesday announced that the U.S. Food and Drug Administration granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy.

"Receiving Orphan Drug designation for KER-065 highlights the significant unmet medical need for patients with DMD," said Jasbir S. Seehra, President and Chief Executive Officer. "This designation serves as a significant milestone for Keros as we advance KER-065 into a Phase 2 clinical trial in patients with DMD."

The FDA grants Orphan Drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States.

KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB that is fused to the portion of the human antibody known as the Fc domain.

Duchenne Muscular Dystrophy (DMD) is the most common form of muscular dystrophy and results in muscle degeneration and premature death. DMD results from the lack of functional dystrophin protein that helps promote myofiber stability, caused by a gene mutation.