European Commission Grants Orphan Designation to Theriva Biologics' VCN-01 for Retinoblastoma

Theriva Biologics, a clinical-stage biotech company developing cancer therapeutics, announced that the European Commission has adopted the EMA’s recommendation to grant orphan medicinal product designation to VCN-01. VCN-01, a systemic, selective, stroma-degrading oncolytic adenovirus for treating retinoblastoma. This designation follows previous recognitions by the U.S. Food and Drug Administration (FDA), which has granted both orphan drug designation and rare pediatric disease designation to VCN-01 for retinoblastoma.

Publish Date: 16-10-2024 Source: Theriva Biologics

Retinoblastoma, although rare, stands as the predominant ocular neoplasm in childhood. It emerges as a malignant proliferation originating from the developing retina, typically manifesting before age five. This tumor arises from cells harboring cancer-predisposing variants in both alleles of the RB1 gene. Its presentation may be unifocal or multifocal. Approximately 60% of cases present as unilateral retinoblastoma, with a mean diagnosis age of 24 months, while around 40% present with bilateral involvement, typically diagnosed at 15 months of age. Heritable retinoblastoma follows an autosomal dominant pattern, increasing susceptibility to the disease and predisposing affected individuals to non-ocular tumors. The prognosis of localized disease is favorable for long-term survival, in stark contrast to metastatic retinoblastoma, characterized by aggressive behavior and dismal outcomes. The genetic underpinning of retinoblastoma is well-established, with evidence suggesting autosomal dominant inheritance, particularly notable in bilateral cases. Retinoblastoma treatment strategies are comprehensive and aim at life-saving and vision-preserving measures. Intraocular disease exhibits high responsiveness to treatment, with over 90% of patients achieving survival. Chemotherapeutic regimens, such as the combination of carboplatin, etoposide phosphate, and vincristine sulfate, capitalize on synergistic effects to enhance efficacy, exploiting distinct mechanisms of action against cancer cells. In the United States, retinoblastoma afflicts approximately 11.8 cases per million live births among children under 5 years old, representing around 2% of all childhood malignancies. The disease predominantly affects infants and toddlers, with an average onset at 2 years of age, while occurrences beyond the age of six are exceedingly rare.

However, the current Retinoblastoma treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Episcleral Topotecan, Conbercept ophthalmic injection, Enzalutamide with Ribociclib, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Retinoblastoma treatment. The key companies in the advanced development stage are Targeted Therapy Technologies, LLC, Chengdu Kanghong Biotech Co., Ltd., Novartis, etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Retinoblastoma to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

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