FDA Orphan Drug Designation Bestowed to VCN-01 Plus Chemotherapy for Early PDAC


The phase 2b VIRAGE study will investigate the safety and efficacy of VCN-01 plus standard chemotherapy in patients with first-line pancreatic ductal adenocarcinoma.

  • With an FDA orphan drug designation (ODD), VCN-01 with gemcitabine and nab-paclitaxel will have the potential benefit of market exclusivity for 7 years, if later granted FDA approval.

  • VCB-01 with gemcitabine and nab-paclitaxel will also be eligible for or tax credits for qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance and potential qualification for expedited development programs, according to the FDA.

  • FDA orphan drug designation was previously granted to VCN-01 plus gemcitabine and nab-paclitaxel for the treatment of retinoblastoma.

Red cancer cells | Image Credit: phonlamaiphoto -www.stock.adobe.com

Image Credit: phonlamaiphoto -www.stock.adobe.com

The FDA has granted an ODD to VCN-01 in combination with the standard chemotherapy doublet of gemcitabine and nab-paclitaxel for the frontline treatment of patients with pancreatic ductal adenocarcinoma (PDAC).1

“The FDA’s decision to grant orphan drug designation to VCN-01 highlights the urgent need for new treatment options for patients with PDAC, which has one of the lowest survival rates among all cancers,” said Steven A. Shallcross, chief executive officer of Theriva Biologics, in a press release. “Efforts to improve upon the standard of care treatment have largely stalled, despite the growing incidence of PDAC, and the need for novel therapies in this indication is acute. The growing clinical data that underscore VCN-01’s multiple modes of action and the compelling clinical outcomes observed in phase 1 studies of VCN-01 in combination with chemotherapy or immunotherapy in patients with PDAC and other solid tumors, give us confidence that VCN-01 has the potential to address this unmet medical need.”

About the VIRAGE Study

Trial Name: A Phase IIb, Open-label, Randomized Study of Nab-Paclitaxel and Gemcitabine and Plus/Minus VCN-01 in Patients With Metastatic Pancreatic Cancer

ClinicalTrials.gov Identifier: NCT05673811

Sponsor: Theriva Biologics SL

Recruitment Contact: Michael Kaleko, MD, PhD, +1 3014174364, mkaleko@therivabio.com

Completion Date: April 28, 2025

VCN-01 is a genetically modified adenovirus being developed for the treatment of pancreatic cancer. In preclinical PDAC models, treatment with the oncolytic adenovirus demonstrated direct antitumor efficacy and degraded the tumor stroma which serves as a barrier to cancer treatment.2 The agent is now under investigation in the phase 2b VIRAGE study (NCT05673811).1,3

VIRAGE follows an open-label, randomized, 2-parallel arm design to evaluate standard-of-care (SoC) treatment with or without VCN-01 in approximately 96 patients with pancreatic cancer.2 Patients will be randomized 1:1 to the SoC arm or the experimental combination arm. In the SoC arm, nab-paclitaxel 125 mg/m2 will be administered as a 30 to 40-minute intravenous (IV) infusion along with gemcitabine 1000 mg/m2 as a 30-minute IV infusion after nab-paclitaxel. SoC treatment will be administered on days 1, 5, and 15 of each 28-day cycle. In the experimental arm, SoC treated at matching doses and schedules as in the comparator arm will be given with VCN-01 1xE13 vp/patient in a single IV infusion lasting 19 minutes on day 1 of the first cycle, followed by day 1 of the 4th cycle.3

The coprimary end points to be explored in VIRAGE include overall survival and the incidence of patients with adverse events. The secondary end points are time to progression, overall response rate, disease control rate, 1-year survival, progression-free survival, duration of response, and changes in tumor marker Ca 19.9.

Both male and female patients who are 18 years of age or older are eligible to enroll in the study. The requirements for inclusion are histologically or cytologically confirmed disease with an ECOG performance score of 0 or 1, and adequate organ function at baseline. Patients excluded from the study are those with active infection, serious illness, autoimmune disease, or other conditions that may interfere with the effectiveness and/or safety of study treatment. Patients who received prior treatment with another investigational agent, live attenuated vaccines within the last 3 weeks, or are receiving full-dose anticoagulant therapy are also ineligible to enroll in the study.

Adult patients with PDAC who meet the eligibility requirements are being recruited at sites in California, Kentucky, New York, Utah, and Spain.


1. 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 June 28, 2023. https://tinyurl.com/3vrbx8ak

2. Bazan-Peregrino M, Garcia-Carbonero R, Laquente B, et al. VCN-01 disrupts pancreatic cancer stroma and exerts antitumor effects. J Immunother Cancer. 2021;9(11): e003254. doi: 10.1136/jitc-2021-00325

3. Study of nab-paclitaxel and gemcitabine and plus/minus vcn-01 in patients with metastatic pancreatic cancer (VIRAGE). ClincalTrials.gov. Updated June 27, 2023. Accessed June 28, 2023. https://tinyurl.com/mrxmnp7c

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