UV1 Receives Orphan Drug Designation From EMA in Mesothelioma


The European Medicines Agency has granted orphan drug designation to UV1 in patients with mesothelioma.

Lung Cancer- stock.adobe.com

Lung Cancer- stock.adobe.com

The cancer vaccine UV1 has been granted orphan drug designation by the European Medicines Agency (EMA) for the treatment of patients with mesothelioma.1

“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,” Carlos de Sousa, MD, MBA, CEO of Ultimovacs, said in a press release. “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 orphan drug designation was supported by findings from the phase 2 NIPU trial (NCT04300244) which were presented during the 2023 ESMO Congress. At a median follow-up of 17.3 months (95% CI, 15.8-22.9), data from the first survival analysis demonstrated that patients who received UV1 with ipilimumab (Yervoy) and nivolumab (Opdivo; n = 59) achieved a median overall survival (OS) of 15.4 months (95% CI, 11.1-22.6) compared with 11.1 months (95% CI, 8.8-18.1) among patients treated with ipilimumab and nivolumab alone (n = 59; HR, 0.73; 80% CI, 0.53-1.0; 95% CI, 0.45-1.18; P = .197).2

The open-label NIPU study randomly assigned patients with malignant pleural mesothelioma following progression on standard frontline platinum-based chemotherapy 1:1 to receive ipilimumab 1 mg/kg every 6 weeks and nivolumab 240 mg every 2 weeks with or without 8 UV1 vaccinations. Treatment in both arms continued until disease progression, unacceptable toxicity, or for up to 2 years. The primary end point was progression-free survival (PFS); secondary end points included OS, objective response rate (ORR), disease-control rate, and toxicity.

The baseline patient characteristics were well balanced between the 2 arms; the median age in the UV1 arm was 71 years (range, 39-79) compared with 72 years (range 42-83) in the ipilimumab and nivolumab alone arm. Most patients in both arms were male (76.3% vs 79.7%), had an ECOG performance status of 1 (71.2% vs 69.5%), had epithelioid histology (74.6% vs 79.7%), and had a PD-L1 status of less than 1 (52.5% vs 54.2%).

Additional findings from the study showed that NIPU did not meet its primary end point; at a median follow-up of 12.5 months (95% CI, 9.7-15.6), the median PFS was 4.2 months (95% CI, 2.9-9.8) in the investigational arm compared with 4.7 months (95% CI, 3.9-7.0) in the control arm (HR, 1.01; 80% CI 0.75-1.36, 95% CI, 0.64-1.59; P = .979). However, patients in the investigational arm experienced a benefit in terms of ORR vs those in the control arm; the ORR was 31% vs 16%, respectively (OR, 2.4; 95% CI, 1.0-6.3).

Notably, UV 1 plus ipilimumab and nivolumab displayed a comparable safety profile with that of ipilimumab and nivolumab alone. Investigators noted that the total number of adverse effects (AEs) was comparable between the 2 arms, as was the proportion of patients who experienced an AE of grade 3 or higher severity.

UV1 in combination with ipilimumab and nivolumab previously received fast track designation from the FDA on February 5, 2024, for the treatment of patients with unresectable malignant pleural mesothelioma. The indication was granted based on improved OS findings from NIPU.3

“We are pleased that the FDA has granted Fast Track designation for UV1 in two separate advanced indications, which underlines the potential of our cancer vaccine approach. UV1 demonstrated a positive safety profile and encouraging signs of improvement in OS in combination with the checkpoint inhibitors, ipilimumab, and nivolumab, in malignant mesothelioma, a hard-to-treat cancer indication with significant unmet need,” de Sousa, said in a press release at the time. “We expect to announce topline results from our randomized phase II trial INITIUM [(NCT04382664] in the coming month of March, and we are looking forward to reporting important data from our broad UV1 phase II clinical trial program with UV1 over the course of 2024 and beyond.”


  1. Ultimovacs receives EMA orphan drug designation for UV1 cancer vaccine for the treatment of mesothelioma. News release. Ultimovacs ASA. February 19, 2024. Accessed February 19, 2024. https://finance.yahoo.com/news/ultimovacs-receives-ema-orphan-drug-060000956.html#:~:text=Oslo%2C%20February%2019%2C%202024%3A,Designation%20(ODD)%20for%20its%20therapeutic
  2. Helland, Haakensen VD, Ojlert AK, et al. First survival data from the NIPU trial: A randomised, open-label, phase II study evaluating nivolumab and ipilimumab combined with UV1 vaccination as second-line treatment in patients with malignant mesothelioma. Ann Oncol. 2023;34(suppl 2):1337. doi:10.1016/j.annonc.2023.10.102
  3. Ultimovacs receives FDA fast track designation for UV1 cancer vaccine for the treatment of patients with unresectable mesothelioma. News release. Ultimovacs ASA. February 5, 2024. Accessed February 19, 2024. https://finance.yahoo.com/news/ultimovacs-receives-fda-fast-track-060000983.html
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