SEATTLE, WA — Atossa Therapeutics, Inc. (Nasdaq: ATOS) (“Atossa” or the “Company”), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of high unmet clinical need, today announced that the U.S. Food and Drug Administration (“FDA”) Office of Orphan Products Development (“OOPD”) has granted Orphan Drug Designation to (Z)-endoxifen for the treatment of Duchenne muscular dystrophy (“DMD”).
“In addition to the previously received Rare Pediatric Disease designation, Orphan Drug Designation for (Z)-endoxifen in Duchenne muscular dystrophy is an important milestone for Atossa as we move forward developing (Z)-endoxifen for this serious and debilitating disease,” noted Steven C. Quay, M.D., Ph.D., Atossa Therapeutics President and Chief Executive Officer.
Atossa plans to continue engaging with FDA as it advances development efforts and will provide updates as appropriate.
About Orphan Drug Designation
Orphan Drug Designation is granted by FDA to therapies intended to treat rare diseases or conditions. The designation is designed to encourage drug development by offering certain potential incentives, such as regulatory support and, if the product ultimately receives marketing approval for the designated indication, eligibility for a period of market exclusivity. FDA also notes that if an “otherwise same drug” is approved first for the same indication, a sponsor may need to demonstrate that the new drug is clinically superior to the previously approved drug in order to be eligible for orphan-drug exclusivity. Orphan Drug Designation neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy is a rare, progressive, X-linked neuromuscular disorder caused by mutations in the dystrophin gene. Symptoms typically emerge in early childhood and include progressive muscle weakness, loss of ambulation, respiratory compromise, and cardiomyopathy. DMD is uniformly fatal, often in early adulthood, and despite recent therapeutic advances, there remains a substantial unmet medical need for safe, effective, and accessible treatments.
About (Z)-Endoxifen
(Z)-Endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/SERD) with demonstrated activity across multiple mechanisms of interest. Atossa is evaluating its potential applications in oncology and rare diseases. The Company’s proprietary oral formulation has shown a favorable safety profile and pharmacology distinct from tamoxifen, including ER-targeted effects and PKC inhibition. Atossa’s (Z)-Endoxifen is not approved for any indication.
Atossa’s (Z)-Endoxifen program is supported by a growing global intellectual property portfolio, including multiple recently issued U.S. patents and numerous pending applications worldwide.
About Atossa Therapeutics
Atossa Therapeutics, Inc. (Nasdaq: ATOS) is a clinical-stage biopharmaceutical company developing innovative medicines in oncology and other areas of significant unmet need. The Company’s lead product candidate, (Z)-Endoxifen, is currently in development across several clinical settings. More information is available at https://atossatherapeutics.com.
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