ROCKVILLE, Md. - Theriva Biologics, Inc. (NYSE American: TOVX), a company focused on developing treatments for cancer and related diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Drug Designation (RPDD) to its leading product candidate, VCN-01, for the treatment of retinoblastoma, a rare type of eye cancer in children.
This designation follows the previously awarded orphan drug status for VCN-01 and emphasizes the critical need for new therapies for pediatric retinoblastoma patients. According to Theriva's CEO, Steven A. Shallcross, the company is working with healthcare professionals and regulatory bodies to refine the clinical approach for VCN-01 as a supplementary therapy to chemotherapy for children with advanced stages of the disease.
VCN-01 is an oncolytic adenovirus that targets and destroys cancer cells while breaking down the stroma, a barrier that impedes effective cancer treatment. This therapy has shown promise in a Phase 1 trial, deemed successful by the study Monitoring Committee, which evaluated the safety and activity of VCN-01 in pediatric patients with refractory retinoblastoma.
The RPDD is granted to drugs that address severe, life-threatening conditions affecting fewer than 200,000 individuals in the U.S., primarily those under 18 years of age. With this designation, Theriva may become eligible for a Priority Review Voucher should the FDA approve a Biologics License Application for VCN-01. This voucher could expedite the review process for any subsequent marketing application or be sold or transferred.
Retinoblastoma affects approximately 1 in 14,000 to 18,000 live newborns and represents 15% of tumors in children under one year of age. The average age at diagnosis is two, and it is uncommon in children over six. In the U.S., there are about 200 to 300 new cases each year. Treatment goals include preserving life and preventing blindness or the loss of an eye, which can significantly impact the patient's lifespan and quality of life.
The FDA's decision to grant RPDD to VCN-01 reflects the ongoing commitment to address the unmet medical needs in pediatric oncology. Theriva Biologics continues to advance its clinical programs and aims to improve treatment outcomes for patients with retinoblastoma. This development is based on a press release statement from Theriva Biologics, Inc.
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