Investing.com -- Atossa Therapeutics Inc (NASDAQ:ATOS) stock jumped 13.6% in premarket trading Thursday after the company announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease (RPD) designation to its (Z)-Endoxifen drug candidate for the treatment of Duchenne Muscular Dystrophy (DMD).
The clinical-stage biopharmaceutical company said the designation is an important regulatory milestone that validates the science behind (Z)-Endoxifen’s potential as a treatment for DMD, a serious childhood disease. The RPD status is given to drug candidates targeting serious conditions primarily affecting individuals from birth to 18 years of age.
Upon approval of a qualifying marketing application, drugs with RPD designation may be eligible for a Priority Review Voucher, which can be used to obtain priority review for a future application or sold to another sponsor. Recent Priority Review Voucher sales have ranged from $100 million to $160 million over the past 18-24 months, according to the company.
"DMD is one of the most devastating childhood diseases. Families urgently need better options beyond steroids and gene-targeted approaches," said Steven Quay, President and Chief Executive Officer of Atossa Therapeutics.
The company noted that (Z)-Endoxifen does not target specific exon defects, potentially offering a broader treatment approach for DMD patients compared to existing therapies. While oncology remains Atossa’s core focus, this development highlights the drug’s potential as a platform therapy in both cancer and rare diseases.
Atossa plans to advance the DMD program to clinical trials, with the RPD designation providing a regulatory framework and enhanced interaction with the FDA as the company defines its clinical development path.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.
