Atossa Therapeutics Inc (NASDAQ:ATOS) experienced a 10.3% increase in its share price on Monday after the U.S. Food and Drug Administration granted Rare Pediatric Disease - RPD - designation to (Z)-endoxifen for the treatment of McCune-Albright Syndrome in females.
Under the RPD program, a drug that receives the designation and later gains approval may qualify its sponsor for a Priority Review Voucher - PRV. That voucher can be applied to accelerate the review of another regulatory submission or sold to a third party. The company noted that recently disclosed PRV transactions have fetched roughly $100 million to $205 million over the past 18 to 24 months.
The RPD designation is reserved for drug candidates intended to address serious or life-threatening conditions that predominantly affect individuals from birth through age 18. McCune-Albright Syndrome is characterized in the company's release as a rare pediatric endocrine disorder that impacts young girls through hormone dysregulation and the onset of early puberty.
In commenting on the designation, Steven Quay, President and Chief Executive Officer of Atossa Therapeutics, said: "This designation is an important regulatory milestone for Atossa and provides further validation of the potential of (Z)-endoxifen beyond oncology." The company also reported involvement in the Fibrous Dysplasia, McCune-Albright Syndrome Alliance Research Priorities Workshop hosted at Children’s Hospital of Philadelphia, which gathered clinicians, researchers, patients, caregivers, and advocacy groups.
Janet Rea, Senior Vice President of Research and Development at Atossa, described the RPD recognition as creating a regulatory pathway and additional chances for "enhanced engagement with the FDA as the company evaluates development strategies for MAS." The firm highlighted (Z)-endoxifen’s mechanism of action as a potent selective estrogen receptor modulator/degrader - SERM/D - and its potential to influence estrogen-driven manifestations of disease.
Atossa is identified as a clinical-stage biopharmaceutical company developing therapies in oncology and other areas of unmet clinical need. The RPD designation and potential PRV represent regulatory and commercial elements the company can consider as it advances its development planning for (Z)-endoxifen in McCune-Albright Syndrome.
Contextual notes
- RPD designation - confers eligibility for a Priority Review Voucher upon approval.
- PRV market - recent disclosed sales have been reported in a range approximating $100 million to $205 million in the last 18 to 24 months.
- Clinical focus - McCune-Albright Syndrome primarily affects pediatric patients through endocrine dysfunction and early puberty.