FDA Grants Rare Pediatric Drug Designation to Theriva Biologics' VCN-01
VCN-01 gets FDA's RPDD for retinoblastoma, aiding Theriva Biologics' treatment development.
Breaking News
Aug 01, 2024
Mrudula Kulkarni

The FDA has awarded VCN-01, a rare paediatric drug
designation (RPDD), to TherivaTM Biologics, a diversified clinical-stage
company that develops therapeutics to treat cancer and related diseases in
high-unmet need areas. The FDA made this announcement today. VCN-01 is being
used to treat retinoblastoma. Theriva's primary product candidate, VCN-01, is
an oncolytic adenovirus that selectively degrades stroma and is systemic.
VCN-01 was formerly designated as an orphan medication by the FDA to treat
retinoblastoma.
Theriva Biologics CEO Steven A. Shallcross stated, "The
FDA's decision to grant rare paediatric drug designation to VCN-01 highlights
the urgent need for new treatment options for paediatric patients with
retinoblastoma." "This significant advancement gives us hope, and we
will keep collaborating closely with top medical professionals and government
organisations to improve our clinical approach for VCN-01 as a chemotherapy
adjuvant in paediatric patients with advanced retinoblastoma. The study
Monitoring Committee most recently declared that the findings of the
investigator-sponsored Phase 1 trial, which assessed the efficacy and safety of
intravitreal VCN-01 in paediatric patients with refractory retinoblastoma, were
favourable. The information obtained from this study will help to improve our
clinical development strategy in this highly unmet need sector.
Less than 200,000 people in the US have rare diseases that
are considered severe and potentially fatal, especially affecting children
under the age of 18. The FDA authorises RPDD for certain conditions. Theriva
may be eligible to obtain a Priority Review Voucher if the FDA approves a
Biologics License Application for VCN-01 for the treatment of retinoblastoma.
This voucher can be sold or transferred, or used to get a priority review for
any later marketing application.