Theriva Biologics receives rare pediatric drug designation from FDA for VCN-01 for the treatment of retinoblastoma

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Theriva Biologics Inc announced the FDA has granted Rare Pediatric Drug Designation (RPDD) for VCN-01 for the treatment of retinoblastoma.

According to the company, VCN-01, its lead product candidate, is a systemic, selective, stroma-degrading oncolytic adenovirus. The FDA had previously granted orphan drug designation to VCN-01 for treatment of retinoblastoma.¹

Steven A. Shallcross, CEO of Theriva Biologics, pointed out in a news release 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, in parallel, 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 said in a statement. “Most recently, results from the 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.”

Shallcross added that data from this study will further inform our clinical development pathway in this area of high unmet need.

Moreover, the FDA grants RPDD for rare diseases (fewer than 200,000 affected persons in the United States) that are serious, life-threatening and primarily affect children ages 18 years or younger. If a Biologics License Application for VCN-01 for the treatment of retinoblastoma is approved by the FDA, Theriva may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred.¹

Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 - 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old.

The company noted in its news release the average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In the U.S., retinoblastoma shows an incidence rate of 3.3 per 1,000,000 with only about 200 to 300 children diagnosed per year according to the American Cancer Society.²

Preserving life and preventing the loss of an eye, blindness and other serious effects of treatment that reduce the patient’s life span or the quality of life, remains a challenge. In addition, children with retinoblastoma are more likely to lose their eye and die of metastatic disease in low-resource countries.

According to the company, VCN-01 is designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma which serves as a significant physical and immunosuppressive barrier to cancer treatment. This mode-of-action enables VCN-01 to exert multiple antitumor effects by selectively infecting and lysing tumor cells; enhancing the access and perfusion of co-administered chemotherapy products; and increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products.¹

This systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases.

References:

1. Inc TB. TherivaTM Biologics Receives Rare Pediatric Drug Designation by the U.S. FDA for VCN-01 for the Treatment of Retinoblastoma. GlobeNewswire News Room. Published July 31, 2024. Accessed August 1, 2024. https://www.globenewswire.com/news-release/2024/07/31/2921842/0/en/Theriva-Biologics-Receives-Rare-Pediatric-Drug-Designation-by-the-U-S-FDA-for-VCN-01-for-the-Treatment-of-Retinoblastoma.html

2. Retinoblastoma | Eye Cancer in Children. www.cancer.org. https://www.cancer.org/cancer/types/retinoblastoma.html