VCN-01 Granted Orphan Drug Designation for Retinoblastoma

In a February 8, 2022 news release, late-stage clinical company Synthetic Biologics, Inc. shared that the FDA granted Orphan Drug designation to the therapeutic candidate VCN-01. VCN-01 is a therapeutic from VCN Biosciences, S.L. – though measures are in the works for Synthetic Biologics to acquire VCN Biosciences. While VCN-01 is being explored in relation to pediatric osteosarcoma and pancreatic cancer, this most recent Orphan Drug designation was granted for retinoblastoma, a rare eye cancer. 

About VCN-01

According to Sant Joan de Deu Hospital Barcelona

Oncolytic adenovirus VCN-01 is an experimental virus that replicates only in tumor cells and destroys them.

The hospital page explains that VCN-01 could be used in aggressive cancers which do not respond well to chemotherapy, or that relapse following chemotherapy. Currently, there are a number of clinical trials evaluating VCN-01. So far, share researchers, the available data is promising. In the future, researchers hope to continue evaluating the therapy in more advanced trials.

VCN-01 received Orphan Drug designation from the FDA. This status is granted to drugs or biologics intended to treat, prevent, or diagnose a rare disease or condition. In reference to “rare,” the FDA is referring to any conditions affecting under 200,000 Americans. With this status also comes a variety of incentives from drug developers, including fee waivers, tax credits, increased regulatory assistance, and 7 years market exclusivity upon approval. 


Retinoblastoma is a rare eye cancer which begins in immature cells within the retina. In some cases, there is a genetic component to retinoblastoma; for these diagnoses, the name is “hereditary retinoblastoma.” However, in other cases, the cause is unknown. This cancer may affect one or both eyes. While it can, in rare cases, impact adults, retinoblastoma most commonly affects younger children, manifesting before age 5. Symptoms and characteristics include:

  • Eye redness and inflammation
  • Increased risk of other cancers
  • Eyes that appear to be looking in different directions
  • Poor vision
  • A white color in the pupil when light is shone into it

Learn more about retinoblastoma

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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