Business
Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy
Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease
About this update from Atossa Therapeutics, Inc.
[{"type":"text","content":"Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease DesignationSEATTLE, Jan. 16, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) (\"Atossa\" or the \"Company\"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of high unmet clinical need, today announced that the U.S. Food and Drug Administration (\"FDA\") Office of Orphan Products Development (\"OOPD\") has granted Orphan Drug Designation to (Z)-endoxifen for the treatment of Duchenne muscular dystrophy (\"DMD\").\n \n \n \n \n \n \n \n\"In addition to the previously received Rare Pediatric Disease designation, Orphan Drug Designation for (Z)-endoxifen in Duchenne muscular dystrophy is an important milestone for Atossa as we move forward developing (Z)-endoxifen for this serious and debilitating disease,\" noted Steven C. Quay, M.D., Ph.D., Atossa Therapeutics President and Chief Executive Officer.Atossa plans to continue engaging with FDA as it advances development efforts and will provide updates as appropriate.About Orphan Drug DesignationOrphan Drug Designation is granted by FDA to therapies intended to treat rare diseases or conditions. The designation is designed to encourage drug development by offering certain potential incentives, such as regulatory support and, if the product ultimately receives marketing approval for the designated indication, eligibility for a period of market exclusivity. FDA also notes that if an \"otherwise same drug\" is approved first for the same indication, a sponsor may need to demonstrate that the new drug is clinically superior to the previously approved drug in order to be eligible for orphan-drug exclusivity. Orphan Drug Designation neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process.About Duchenne Muscular DystrophyDuchenne Muscular Dystrophy is a rare, progressive, X-linked neuromuscular disorder caused by mutations in the dystrophin gene. Symptoms typically emerge in early childhood and include progressive muscle weakness, loss of ambulation, respiratory compromise, and cardiomyopathy. DMD is uniformly fatal, often in early adulthood, and despite recent therapeutic advances, there...