Ultimovacs has announced that the European Medicines Agency (EMA) has issued a positive opinion on the company’s application for  Orphan Drug Designation (ODD) for its therapeutic cancer vaccine UV1 for the treatment of mesothelioma.

The designation was granted based on results from the Phase II clinical trial, NIPU evaluating UV1 added to ipilimumab and nivolumab treatment in patients with malignant pleural mesothelioma.

“The EMA’s Orphan Drug Designation for UV1 in mesothelioma is an important step forward in the development of our cancer vaccine in this indication,” says Carlos de Sousa, CEO of Ultimovacs. “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.”

The NIPU trial

The impact of UV1 vaccination in patients with malignant pleural mesothelioma has been evaluated in a randomized Phase II clinical trial, NIPU. In the study, UV1 was combined with checkpoint inhibitors ipilimumab and nivolumab and compared to ipilimumab and nivolumab alone as a second-line treatment, after first-line treatment with platinum-based chemotherapy. The results from the study, presented at the European Society for Molecular Oncology (“ESMO”) Congress 2023, demonstrated a clinically meaningful improvement in overall survival for UV1 treatment in combination with ipilimumab and nivolumab versus ipilimumab and nivolumab alone. In addition, UV1 maintained its positive safety and tolerability profile in this combination with no added toxicities. Oslo University Hospital sponsored the NIPU study with support from Bristol Myers Squibb and Ultimovacs.

UV1

On February 5, 2024, the FDA granted Fast Track designation for UV1 in combination with ipilimumab and nivolumab for the treatment of patients with unresectable malignant pleural mesothelioma to improve overall survival. In October 2023, UV1 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with mesothelioma. In December 2021, the FDA granted Orphan Drug Designation for UV1 for the treatment of stage IIB-stage IV melanoma. In October 2021, the FDA granted Fast Track designation for UV1 as add-on therapy to ipilimumab or pembrolizumab for the treatment of unresectable or metastatic melanoma.

Photo of Carlos de Sousa: Ultimovacs