Theriva Biologics (AMEX: TOVX), a clinical-stage immuno-oncology company, has been granted Rare Pediatric Drug Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for its drug VCN-01, designed to treat retinoblastoma. Retinoblastoma is the most common type of eye cancer in children and poses significant treatment challenges.
The prevalence of cancer among children is rising globally, with an estimated 15,780 children diagnosed with cancer annually in the U.S. alone, according to the American Childhood Cancer Organization. Retinoblastoma, though accounting for only 2% of all childhood cancers, affects 200 to 300 children each year in the U.S., predominantly infants and young children. The average age of diagnosis is two years old.
Retinoblastoma can significantly impact a child's quality of life, often necessitating difficult choices between preserving life and preventing loss of vision or an eye. In lower-resource countries, children face higher risks of losing their eyes and succumbing to metastatic disease.
Market research firm Spherical Insights projects that the global market for retinoblastoma treatment, valued at $2.5 billion in 2023, will grow to $3.8 billion by 2033. This growth underscores the urgent need for effective treatment options for this rare but impactful disease.
The Rare Pediatric Drug Designation is awarded to drugs developed specifically for rare pediatric diseases. This designation encourages the development of medications for conditions that are often overlooked due to their rarity. For Theriva Biologics, this designation offers a significant incentive: if the Biologics License Application for VCN-01 is approved, the company may be eligible for a Priority Review Voucher (PRV). PRVs can be redeemed for priority review of any subsequent marketing application or sold, with previous transactions valued around $100 million.
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. We are encouraged by this important step forward and continue to work closely with leading physicians and regulatory agencies to refine our clinical strategy for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma.”
Shallcross also noted that recent results from a Phase 1 trial evaluating VCN-01 in pediatric patients with refractory retinoblastoma were positive. These findings will help guide the clinical development pathway for this high-need area.
The RPDD milestone for VCN-01 underscores the drug's potential efficacy and long-term impact. It also highlights the broader social benefits Theriva Biologics aims to provide, potentially increasing its influence in the medical and pharmaceutical industries.
