FDA Grants Orphan Drug Designation to VCN-01 for Retinoblastoma Treatment


The novel oncolytic adenovirus, VCN-01 has been granted FDA orphan drug designation for retinoblastoma and will soon be investigated in a phase 2/3 clinical trial.

VCN-01, a genetically modified oncolytic adenovirus has received orphan drug designation by the FDA for the treatment of patients with retinoblastoma (RB), according to a press release by Synthetic Biologics, Inc.1

VCN-01 is a product of VCN, a clinical-stage immuno-oncology company focused on the development of the next generation of oncolytic adenoviruses. They are currently developing a new oncolytic adenovirus (OV) platform designed for intravenous (IV) delivery to trigger tumor cell death and promote immune cell infiltration into tumors.

The OV is designed to target primary and metastatic tumors, replicate selectively and aggressively and to produce hyaluronidase, an enzyme that digests hyaluronan, when inside of the tumor.

Previous completed clinical trials have concluded that this process can occur anywhere from weeks and months following a single IV injection with a VCN OV.

"We are pleased to report that VCN's VCN-01 was granted Orphan Drug Designation for retinoblastoma by the FDA. We believe VCN-01 may represent a novel rescue therapy for patients who fail standard therapy, or may be used as an adjunct to chemotherapy, to provide improved outcomes for these patients.We are highly encouraged by the preliminary clinical data thus far and look forward to conducting a pivotal phase 2/3 trial in these patients following our expected completion of the acquisition," said Steven Shallcross, chief executive officer and chief financial officer of Synthetic Biologics, in the press release.

Treatment with the OVs combined with a variety of chemotherapies and immunotherapies such as checkpoint inhibitors and CAR T cells are currently in early clinical or planned testing.

VCN-01, the lead product of VCN, is an oncolytic adenovirus with unique characteristics being studied in clinical trials for cancers for which there is no cure, including pancreatic carcinoma and RB. VCN-01 has been evaluated in four phase 1 clinical trials to date.

In one trial, patients with metastatic pancreatic ductal adenocarcinoma received the combination therapy of IV VCN-01 and the standard of care chemotherapy gemcitabine plus paclitaxel (G/A) VCN-01 administered one week before the first G/A dose concluded the best results.

When combined with G/A, VCN-01 was well tolerated and demonstrated an improved median overall and progression-free survival, as well as a high response rate when compared to G/A alone.

The findings are compared favorably with current standard of care and are the basis of a planned phase 2 clinical trial focusing on a higher dosing level.

VCN-01 is also being studied as a single therapy in patients with RB who previously failed chemotherapy. Intravitreal administration of VCN-01 produced a complete remission and a reduction of tumors in several patients. These promising outcomes are what created the basis for the now approved orphan drug application.

The FDA's Office of Orphan Products Development is in charge of granting orphan status to developing drugs which treat, diagnose or prevent a rare disease or condition which affect fewer than 200,000 people in the United States. Orphan Drug Designation provides drug developers with various benefits to support the development of novel drugs.

These benefits include the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance and potential qualification for expedited development programs.


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 February 8, 2022. https://bit.ly/3gvqwii

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