Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for DMD Drug (Z)-Endoxifen

Atossa Therapeutics Advances DMD Treatment with FDA Designation

Atossa Therapeutics, Inc., a clinical-stage biopharmaceutical company, achieves a significant milestone by receiving Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA) for its drug candidate, (Z)-Endoxifen. This designation is crucial as it pertains to serious or life-threatening conditions in children and young adults aged 0 to 18, specifically targeting Duchenne Muscular Dystrophy (DMD). This degenerative disease severely impacts muscle function and is characterized by progressive weakness, making effective treatment options critical for affected families. The designation not only underscores the urgency of developing better therapies for DMD but also positions Atossa for a Priority Review Voucher (PRV) upon successful marketing application, a financial incentive that has recently commanded prices between $100 million and $160 million in the market.

Dr. Steven Quay, Atossa's President and CEO, underscores the importance of this milestone, calling it a validation of (Z)-Endoxifen's potential to fill a significant gap in DMD treatment options. Unlike existing therapies that target specific exon defects in the dystrophin gene, (Z)-Endoxifen takes a more comprehensive approach, which could enhance accessibility for a broader range of patients. This innovative mechanism positions Atossa uniquely in the competitive landscape of DMD therapies, suggesting a paradigm shift in how the disease may be managed. The company is eager to leverage this designation to accelerate the clinical development of (Z)-Endoxifen, aiming to bring a new treatment option to market for those affected by this debilitating condition.

Janet Rea, Senior Vice President of Research and Development at Atossa, highlights the advantages of the RPD designation, particularly in fostering enhanced interactions with the FDA as the company advances its clinical strategy. Encouraged by promising preclinical data that support (Z)-Endoxifen’s role as a potent Selective Estrogen Receptor Modulator/D (SERM/D), Atossa is poised to move forward with its clinical trials. The company has previously obtained Investigational New Drug (IND) clearance for another DMD treatment, Exondys 51® (eteplirsen), which reinforces its commitment to addressing the needs of patients with rare diseases. By expanding its therapeutic portfolio beyond oncology, Atossa aims to create non-dilutive value through active participation in the Rare Pediatric Disease program.

Atossa’s recent developments reflect a strategic pivot towards addressing rare diseases, an area of unmet medical need that aligns with the company’s mission and growth objectives. By focusing on innovative treatments like (Z)-Endoxifen, Atossa is not only advancing scientific research but also fostering hope for families impacted by DMD. As the company progresses through regulatory hurdles and clinical trials, it stands at the forefront of a transformative approach to pediatric health care, potentially reshaping the landscape of DMD treatment.