FDA Grants VCN-01 Rare Pediatric Disease Designation in Retinoblastoma

Microscopic rendering of oncolytic virus - Generated with Google Gemini AI

• The FDA has granted rare pediatric disease designation (RPDD) to VCN-01 for the treatment of retinoblastoma.
• VCN-01 is a stroma-degrading oncolytic adenovirus.
• VCN-01 was previously granted orphan drug designation from the FDA for the same indication.

VCN-01, a selective, stroma-degrading, oncolytic adenovirus, has been granted RPDD by the FDA for the treatment of retinoblastoma.¹

The RPDD program intends to incentivize drug development for rare pediatric disease. If the FDA approves a biologics license application of VCN-01, the sponsor may receive a priority review voucher that it can use on another product, sell, or transfer to another sponsor.

“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,” said Steven A. Shallcross, chief executive officer of Theriva Biologics, in a press release. “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.”

In 2022, the FDA granted VCN-01 an orphan drug designation for the same indication.² Additionally, VCN-01 in combination with gemcitabine and nab-paclitaxel was granted a fast track designation and an orphan drug designation in pancreatic cancer.^3,4

About VCN-01

VCN-01 is a systemically administered oncolytic adenovirus treatment. It works by selectively infecting and lysing tumor cells as well as enhancing access and perfusion of any coadministered chemotherapy agents. The use of this oncolytic agent can also increase a tumor’s immunogenicity, improving exposure to any coadministered immunotherapy agents.

A phase 1 study (NCT03284268) is investigating VCN-01 in patients with retinoblastoma.5 Patients are receiving 1 of 3 dose levels as an intravitreal injection. The primary end point of the study is incidence of treatment-emergent adverse events, and secondary end points include tumor and immune responses.

Patients aged 1 to 12 with relapsed or refractory retinoblastoma who have normal renal, hepatic, and marrow functions are eligible for participation in the study. Those with uncontrolled epilepsy, cardiac disease, glaucoma, active infections, or immunosuppression are not eligible for study enrollment.

REFERENCES:

1. Theriva™ Biologics receives rare pediatric drug designation by the U.S. FDA for VCN-01 for the treatment of retinoblastoma. News release. Theriva Biologics, Inc. July 31, 2024. Accessed August 1, 2024. https://tinyurl.com/5ehz6f7p

2. Synthetic Biologics announces VCN Biosciences' VCN-01 receives orphan drug designation for retinoblastoma from the U.S. FDA. News release. Synthetic Biologics. February 8, 2022. Accessed August 1, 2024. https://bit.ly/3gvqwii

3. Theriva Biologics announces fast track designation granted by the U.S. FDA for VCN-01 for the treatment of metastatic pancreatic cancer. News release. Theriva Biologics. May 23, 2024. Accessed August 1, 2024. https://tinyurl.com/3tetxdy2

4. Theriva Biologics announces orphan drug designation granted by the U.S. FDA for VCN-01 for the treatment of pancreatic cancer. News release. Theriva Biologics. June 27, 2023. Accessed August 1, 2024. https://tinyurl.com/3vrbx8ak

5. Evaluate safety and the oncolitic adenovirus VCN-01 activity in patients with refractory retinoblastoma (RTB). ClinicalTrials.gov. Updated April 22, 2024. Accessed August 1, 2024. https://clinicaltrials.gov/study/NCT03284268