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Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy

On Jan. 16, 2026, Atossa Therapeutics, Inc. said the FDA Office of Orphan Products Development granted Orphan Drug Designation to -Endoxifen for Duchenne muscular dystrophy in a Seattle press release.
Duchenne muscular dystrophy is a rare, progressive X-linked disorder with early childhood symptoms and a fatal course, while the FDA program encourages rare disease drug development with regulatory support and potential exclusivity.
Atossa's lead candidate, the program, features a proprietary oral formulation with a favorable safety profile distinct from tamoxifen and is supported by a growing global IP portfolio with recently issued U.S. patents.
Atossa said it will continue engaging with the FDA as it advances development, with Steven C. Quay calling the designation an important milestone, while FDA guidance notes exclusivity requires clinical superiority and review time is not shortened.
Atossa is a clinical-stage biopharma developing oncology and rare-disease candidates, and its press release contains forward-looking statements subject to risks that could affect outcomes.

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Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy

Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation

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