Ultimovacs announced in February 2024 that the European Medicines Agency (EMA) has issued a “positive opinion” on its application for Orphan Drug Designation (ODD) for a therapeutic cancer vaccine for the treatment of mesothelioma: UV1. The designation was announced shortly after the US FDA granted Fast Track designation, also in February 2024. These awards were based on results from a Phase II clinical trial to evaluate UV1 in combination with ipilimumab and nivolumab in patients with malignant pleural mesothelioma.
EU ODD and mesothelioma
To qualify for orphan designation, a medicine in the EU must meet several criteria identified by the EMA:
- It must be intended for the treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating
- The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that the marketing of the medicine would generate sufficient returns to justify the investment needed for its development
- No satisfactory method of diagnosis, prevention, or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition
Mesothelioma is a “rare and aggressive” form of cancer with a high mortality rate and limited therapeutic options. Patients with mesothelioma “commonly have a history of occupational or environmental exposure to asbestos”. It usually takes decades for the cancer to develop.
How does UV1 help?
In October 2023 Ultimovacs announced that data from a study that showed the combination approach of UV1 and ipilimumab and nivolumab demonstrated a “statistically significant and clinically meaningful improvement” in overall survival in comparison with ipilimumab and nivolumab alone. Furthermore, UV1 “maintained its positive safety and tolerability profile”.
Dr Carlos de Sousa, Ultimovacs’ CEO, commented that the ODD is an “important step forward in the development of our cancer vaccine in this indication”.
“It allows for important regulatory and commercial benefits and provides us with the potential to rapidly advance UV1 for a patient population with poor prognosis and a high unmet medical need.”
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