Theriva Biologics has reached a significant milestone in the treatment of retinoblastoma after obtaining orphan drug designation from the European Commission for its lead clinical candidate, VCN-01. This oncolytic adenovirus, which had previously been recognized by the U.S. Food and Drug Administration (FDA) as a rare and pediatric orphan drug, represents a renewed hope for fighting this type of eye cancer that predominantly affects young children, who have had limited therapeutic options until now.
The company’s CEO, Steven A. Shallcross, expressed satisfaction with this recognition, emphasizing the urgency of developing alternatives to treat retinoblastoma. Despite its rarity, this disease constitutes a notable percentage of cancers affecting children under one year old, making it crucial to search for more effective options. The orphan drug designation for VCN-01 underscores its promising characteristics, evidenced in initial clinical trials that have shown both safety and activity when administered intravitreally in patients with refractory forms of retinoblastoma.
What sets VCN-01 apart is its ability to replicate within tumor cells while degrading the tumor stroma barrier. This allows for more effective access to cancer cells and enhances the efficacy of other therapies, making it a potential complement to standard chemotherapy in retinoblastoma treatment. This cancer is associated with high mortality rates in countries with fewer resources due to the lack of effective treatments.
The European orphan drug designation not only highlights the potential effectiveness of VCN-01, but also provides Theriva Biologics with various benefits, including regulatory assistance and market exclusivity for a decade after approval. The company reaffirms its commitment to closely collaborate with regulatory authorities and medical experts globally, with the intention of optimizing its clinical strategy and advancing the development of this innovative therapy. The goal is to provide a viable and effective response to this devastating childhood cancer, filling a significant gap in current treatment options.
Referrer: MiMub in Spanish
