Atossa gains on Rare Pediatric Disease status for Duchenne asset

 Shares of Atossa Therapeutics (ATOS) rose 10% on Thursday after the company said the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to its investigational therapy (Z)-Endoxifen for Duchenne Muscular Dystrophy.

The company said the designation expands the program into rare pediatric neuromuscular disease and may qualify Atossa for a Priority Review Voucher (PRV) upon future approval. 

The Rare Pediatric Disease designation is reserved for serious or life-threatening conditions affecting individuals from birth to 18 years old. Atossa noted that FDA authority to award new vouchers may lapse unless renewed, with pending U.S. legislation seeking to extend the program through 2029.

Last week, Atossa completed a Type C meeting with the FDA to review regulatory strategy for advancing (Z)-Endoxifen across metastatic disease, neoadjuvant treatment and breast cancer risk-reduction settings. The FDA provided feedback on expedited regulatory pathways, clinical development design and endpoint strategy that the company said could support a streamlined registrational approach.

CEO Steven Quay said the meeting allowed the company to incorporate FDA input into development planning and could shorten its regulatory timeline. Rea said the clinical program is now supported by data from nearly 800 participants enrolled across multiple trials.

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