Atossa Therapeutics Receives FDA Orphan Drug Designation for DMD Treatment (Z)-endoxifen

Atossa Therapeutics Advances Rare Disease Treatment with FDA Designation

Atossa Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on innovative therapies for oncology and urgent medical needs, receives Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its investigational drug (Z)-endoxifen. This designation, which complements a previously granted Rare Pediatric Disease designation, highlights Atossa's commitment to addressing Duchenne muscular dystrophy (DMD), a severe and progressive neuromuscular disorder that primarily affects children. DMD leads to significant muscle weakness and early mortality, creating an urgent need for effective therapeutic options.

The recognition from the FDA emphasizes the critical unmet medical need for DMD treatments, as the disease is caused by mutations in the dystrophin gene. Atossa's (Z)-endoxifen, a potent Selective Estrogen Receptor Modulator/Degrader (SERM/SERD), is still under development and has not yet received approval for any indication. The Orphan Drug Designation provides potential advantages for Atossa, including regulatory support and market exclusivity once approved, which are crucial for the development of treatments targeting rare diseases like DMD. While this designation does not expedite the drug development process, it affirms the significance of Atossa's ongoing research efforts.

Atossa's President and CEO, Steven C. Quay, M.D., Ph.D., emphasizes the company's dedication to overcoming the challenges posed by DMD. The proprietary oral formulation of (Z)-endoxifen has shown a favorable safety profile and distinct pharmacological properties compared to tamoxifen, suggesting its potential in treating both oncology and rare diseases. As Atossa continues to engage with the FDA regarding (Z)-endoxifen's development, the company remains optimistic about the future of its therapeutic pipeline and the possibility of providing meaningful solutions for patients suffering from DMD.

In addition to its efforts in DMD, Atossa's focus on oncology remains strong. The company's innovative approaches to drug development position it to potentially address various critical health issues, reflecting a broader commitment to tackling pressing medical needs. As the clinical landscape evolves, Atossa Therapeutics is poised to make impactful contributions to both rare and prevalent diseases through its research and development initiatives.