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Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy
Designation expands (Z)-Endoxifen program into rare pediatric neuromuscular disease and may qualify Atossa for a future Priority Review Voucher upon
About this update from Atossa Therapeutics, Inc.
[{"type":"text","content":"Designation expands (Z)-Endoxifen program into rare pediatric neuromuscular disease and may qualify Atossa for a future Priority Review Voucher upon approvalSEATTLE, Dec. 11, 2025 /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\") has granted Rare Pediatric Disease (\"RPD\") designation to (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy (\"DMD\").\n \n \n \n \n \n \n \nRPD designation is granted to drug candidates intended to treat serious or life-threatening diseases that primarily affect individuals from birth to 18 years of age. Upon approval of a qualifying marketing application, drugs with RPD designation may be eligible for a Priority Review Voucher (\"PRV\"), which can be used to obtain priority review for a future application or may be sold or transferred to another sponsor. In the last 18–24 months, disclosed PRV sales have ranged from $100–$160 million.\"This designation is an important regulatory milestone for Atossa, and we believe a strong validation of the science supporting the potential of (Z)-Endoxifen as a treatment for Duchenne Muscular Dystrophy,\" said Steven Quay, M.D., Ph.D., Atossa Therapeutics President and Chief Executive Officer. \"DMD is one of the most devastating childhood diseases. Families urgently need better options beyond steroids and gene-targeted approaches. While oncology remains our core focus, this milestone highlights (Z)-Endoxifen's potential as a platform therapy in both cancer and rare diseases, opening the door to potential non-dilutive value creation through the Rare Pediatric Disease program.\"\"RPD designation provides a regulatory framework and an enhanced level of interaction with the FDA as we define the clinical development path in DMD,\" said Janet Rea, MSPH, Senior Vice President of Research and Development at Atossa. \"We are very encouraged by emerging preclinical data and by (Z)-Endoxifen's potential to be a differentiated mechanism as a potent SERM/D, and look forward to our planned advancement of this program to the clinic for boys living with DMD. Unlike more recent therapeutic approaches, (Z)-Endoxifen does not target speci...