Theriva Biologics Receives FDA's Rare Pediatric Drug Designation for Retinoblastoma Treatment
TL;DR
Theriva Biologics gains competitive advantage with Rare Pediatric Drug Designation for VCN-01, potentially receiving a Priority Review Voucher worth $100M.
VCN-01, a drug developed by Theriva Biologics, is granted Rare Pediatric Drug Designation by the FDA for treating retinoblastoma in children.
Theriva Biologics' VCN-01, with Rare Pediatric Drug Designation, aims to provide new treatment options for pediatric patients with retinoblastoma, addressing an urgent unmet need.
Global retinoblastoma treatment market is expected to grow to $3.8 billion by 2033, creating opportunities for innovation and advancements in pediatric cancer treatment.
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Theriva Biologics (AMEX: TOVX), a clinical-stage immuno-oncology company, has been awarded the Rare Pediatric Drug Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for its drug VCN-01, intended to treat retinoblastoma. Retinoblastoma is the most common type of eye cancer in children, typically affecting infants and young children.
According to the American Childhood Cancer Organization, approximately 15,780 children are diagnosed with cancer annually in the U.S. Retinoblastoma accounts for about 2% of these childhood cancers, with 200 to 300 new cases each year. The disease presents significant treatment challenges, including the risk of blindness and other severe consequences that can affect life quality and lifespan.
The RPDD is a special status given to drugs developed specifically for rare pediatric diseases, encouraging the development of treatments for conditions affecting a small number of patients. If Theriva Biologics' Biologics License Application for VCN-01 is approved, the company may be eligible for a Priority Review Voucher (PRV). PRVs can be redeemed for a priority review of any subsequent marketing application or sold, with previous vouchers fetching 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." Shallcross also noted positive results from an investigator-sponsored Phase 1 trial evaluating the safety and activity of VCN-01 in pediatric patients with refractory retinoblastoma, which will inform further clinical development.
The global retinoblastoma treatment market, valued at $2.5 billion in 2023, is projected to grow to $3.8 billion by 2033, according to market research firm Spherical Insights. This growth underscores the increasing demand for effective treatments for this rare and challenging disease.
The designation of VCN-01 as a rare pediatric drug by the FDA marks a significant milestone for Theriva Biologics and highlights the potential of the drug to address an unmet medical need. The long-term social benefits and industry impact of this development could be substantial, offering hope to families affected by retinoblastoma and setting a precedent for future pediatric cancer treatments.
Featured photo by Ani Kolleshi on Unsplash.
Curated from News Direct
