Keros Announces U.S. FDA Orphan Drug Designation Granted to KER-065 for the Treatment of Duchenne Muscular Dystrophy

Core Viewpoint - Keros Therapeutics has received Orphan Drug designation from the FDA for KER-065, aimed at treating Duchenne muscular dystrophy (DMD), highlighting the significant unmet medical need in this area [1][2]. Company Overview - Keros Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel therapeutics targeting disorders linked to dysfunctional signaling of the TGF-ß protein family [5]. - The company is recognized for its expertise in the TGF-ß family of proteins, which are crucial for the growth, repair, and maintenance of various tissues, including blood, bone, skeletal muscle, adipose, and heart tissue [5]. Product Information - KER-065 is a novel ligand trap designed to inhibit the biological effects of myostatin and activin A, promoting skeletal muscle regeneration, increasing muscle size and strength, reducing body fat, and enhancing bone strength [3]. - The product is initially focused on treating DMD, a severe condition characterized by muscle degeneration due to the lack of functional dystrophin protein [4]. Disease Context - Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy, leading to muscle degeneration and premature death, affecting approximately one in every 3,500 male births worldwide [4]. - The absence of dystrophin results in increased susceptibility of muscle cells to damage, progressive muscle cell death, and replacement with fibrotic and fatty tissue, ultimately causing loss of muscle strength and function [4]. Regulatory Milestone - The Orphan Drug designation provides Keros with several benefits, including tax credits for clinical testing, waivers or reductions in FDA application fees, and seven years of market exclusivity upon approval [2].