Theriva Biologics Receives FDA Rare Pediatric Drug Designation for Retinoblastoma Treatment

Theriva Biologics (AMEX: TOVX), a clinical-stage immuno-oncology company, has received Rare Pediatric Drug Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for its drug candidate VCN-01 in the treatment of retinoblastoma. This designation marks a significant milestone in addressing the urgent need for new treatment options for pediatric patients with this rare form of eye cancer.

Retinoblastoma, accounting for approximately 2% of all childhood cancers, affects 200 to 300 children annually in the United States. The cancer typically manifests in infants and young children, with an average diagnosis age of two years. While the incidence is relatively low, the challenges in treating retinoblastoma are substantial, as physicians must balance preserving life with preventing vision loss and other severe consequences that can impact both lifespan and quality of life.

The RPDD granted to VCN-01 is part of a broader initiative to encourage the development of medications for rare pediatric conditions. This designation comes with potential benefits for Theriva Biologics, including eligibility for a Priority Review Voucher (PRV) if the FDA approves a Biologics License Application for VCN-01 in retinoblastoma treatment. PRVs have historically been valued at around $100 million, providing a significant incentive for companies developing treatments for rare pediatric diseases.

Steven A. Shallcross, CEO of Theriva Biologics, emphasized the importance of this designation, stating, "The FDA's decision to grant rare pediatric drug designation to VCN-01 highlights the urgent need for new treatment options for pediatric patients with retinoblastoma." Shallcross also noted that recent results from an investigator-sponsored Phase 1 trial evaluating the safety and activity of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma were determined to be positive by the study Monitoring Committee.

The global retinoblastoma treatment market, valued at $2.5 billion in 2023, is expected to grow to $3.8 billion by 2033, according to market research firm Spherical Insights. This growth projection underscores the increasing demand for effective treatments in this field.

The development of VCN-01 for retinoblastoma aligns with the broader trend of increasing childhood cancer rates. The American Childhood Cancer Organization reports that an estimated 15,780 children between birth and 19 years of age are diagnosed with cancer each year in the U.S. Globally, over 300,000 children are diagnosed with cancer annually, with rates slowly rising from 165 per million in 2003 to 177 per million in 2019.

As Theriva Biologics continues to advance VCN-01 through clinical development, the RPDD serves as a recognition of the potential impact this treatment could have on pediatric patients with retinoblastoma. The company's focus on this rare pediatric cancer demonstrates a commitment to addressing unmet medical needs in vulnerable populations, potentially offering new hope to children and families affected by this challenging disease.