Keros Therapeutics today announced the U.S. Food and Drug Administration (“FDA”) granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (“DMD”).
“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. This designation provides certain potential benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.
Read More: Phase 1 Clinical Trial of KER-065 in Healthy Volunteers
What is KER-065?
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. KER-065 is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin A, two ligands that signal through activin receptors, to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength.
