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Atossa Therapeutics Achieves FDA Rare Pediatric Disease Designation for (Z)-Endoxifen

Atossa Therapeutics (Nasdaq: ATOS), a biopharmaceutical firm focused on innovative therapies, has recently announced its receipt of the Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for its drug candidate, (Z)-Endoxifen. This drug is aimed at treating Duchenne Muscular Dystrophy (DMD), a serious neuromuscular condition mainly affecting boys and characterized by progressive muscle weakness. This designation is a critical milestone for Atossa, as it not only validates their scientific approach but also provides potential avenues for expedited regulatory processes.

The FDA awards the Rare Pediatric Disease designation to medicines that address serious or life-threatening diseases that primarily affect individuals from birth to 18 years of age. Receiving this designation means that once a qualifying marketing application is approved, Atossa may be eligible for a Priority Review Voucher (PRV). This voucher can grant expedited review for a future application or can even be sold or transferred to another company, with past PRV sales reaching as high as $160 million in some cases over the last 18-24 months.

Steven Quay, M.D., Ph.D., Atossa's President and CEO, expressed the significance of this designation, stating, "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. DMD is one of the most devastating childhood diseases. Families urgently need better options beyond steroids and gene-targeted approaches."

While Atossa has primarily focused on oncology, this milestone underscores the capacity of (Z)-Endoxifen to function as a therapeutic platform not only in cancer but also in rare diseases. Janet Rea, MSPH, Senior Vice President of Research and Development at Atossa, elaborated that the RPD designation provides a structured regulatory framework and an enhanced dialogue with the FDA, which is crucial for planning the clinical development pathway for DMD. Rea added, "Emerging preclinical data is encouraging, especially regarding (Z)-Endoxifen's potential as a differentiated mechanism with potent SERM/D activities."

Unlike some recent treatment strategies that target specific defects in the DMD gene, (Z)-Endoxifen offers a broader treatment approach. This is especially vital for a patient population that has limited existing options. Importantly, Atossa has a history of advancing drugs in the DMD space, having previously secured IND clearance for Eteplirsen, a therapy currently used in the DMD treatment landscape.

Understanding Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy is a progressive X-linked neuromuscular disorder resulting from mutations in the dystrophin gene, presenting primarily in early childhood. Patients exhibit progressive muscle weakness, loss of mobility, respiratory issues, and heart complications. Unfortunately, DMD is typically fatal in early adulthood, and despite some recent therapeutic advancements, there remains a compelling demand for safe, efficient, and accessible treatment options.

The Role of (Z)-Endoxifen

(Z)-Endoxifen is categorized as a Selective Estrogen Receptor Modulator/Degrader (SERM/D) with potential applicability in various therapeutic areas. Atossa is assessing its functions not only in oncology but also in rare diseases. The company believes that its proprietary oral formulation has a favorable safety profile and pharmacological benefits distinct from tamoxifen, including effective ER-targeting and PKC inhibition.

Atossa Therapeutics continues to build a robust global patent portfolio to support (Z)-Endoxifen's program, with numerous recently issued U.S. patents and pending applications worldwide. As progress is made, the company strives to advance its pipeline, ensuring that families affected by DMD have more options for treatment and improved quality of life.

In conclusion, the FDA's Rare Pediatric Disease designation for (Z)-Endoxifen is a notable step forward in addressing the critical need for innovative therapies in the DMD community. The team at Atossa is optimistic about the potential of their research and remains committed to bringing forward solutions that can make a significant difference in the lives of patients and their families.