FDA Grants Orphan Drug Status to Ultimovacs' UV1 Cancer Vaccine for Mesothelioma Treatment

The orphan drug designation was granted based on data from the randomized Phase II clinical trial NIPU

Ultimovacs ASA , a clinical-stage biotechnology leader in novel immunotherapeutic cancer vaccines, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the company’s therapeutic cancer vaccine UV1 for the treatment of patients with mesothelioma. The designation was granted based on the initial data from the Phase II clinical trial, NIPU.

Mesothelioma is a rare and aggressive form of cancer with a high mortality rate and few therapeutic options. Patients with mesothelioma commonly have a history of occupationally or environmentally exposure to asbestos, and it typically takes decades for this specific form of cancer to develop.

The impact of UV1 vaccination in patients with malignant pleural mesothelioma is being assessed in the 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 randomized, open-label, multicenter trial with 118 patients was conducted in Australia, Denmark, Norway, Spain, and Sweden. The first patient in the trial was enrolled in June 2020, and the last patient was enrolled in January 2023. The NIPU study is sponsored by Oslo University Hospital with support from Bristol-Myers Squibb and Ultimovacs.

The results from the study will be shared at the ESMO Congress in Madrid, held October 20-24, in an oral presentation by the Principal Investigator, Aslaug Helland, MD, Ph.D., Professor at Oslo University Hospital. The presentation title is “LBA99 – First survival data from the NIPU trial; A randomized, open-label, phase II study evaluating nivolumab and ipilimumab combined with UV1 vaccination as second-line treatment in patients with malignant mesothelioma”.

“Gaining FDA orphan drug designation for UV1 in mesothelioma highlights UV1’s potential and the significant need for new treatment options for this patient population,” said Carlos de Sousa, CEO of Ultimovacs. “We look forward to the presentation of the NIPU results at the ESMO Congress later this month and to continue our dialogue with the FDA as we seek to bring UV1 to cancer patients as quickly as possible.”

The FDA’s Office of Orphan Products Developments grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including potentially up to seven years of market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.

UV1 is a therapeutic cancer vaccine that generates an immune response against the human telomerase (hTERT) enzyme.  The enzyme is essential for the ability of cancer cells to proliferate. Telomerase is present in 85-90% of all cancers across all stages of the disease.

Ultimovacs is evaluating the universal cancer vaccine UV1 in a broad clinical development program across various cancer indications with different biologies and disease stages, combined with different checkpoint inhibitors. The topline data from NIPU are the first results among the currently five randomized trials in the UV1 Phase II clinical program. In addition to malignant pleural mesothelioma, Phase II studies are ongoing in patients with malignant melanoma, head and neck cancer, ovarian cancer, and non-small cell lung cancer. The topline data from the malignant melanoma and head and neck cancer trials are also expected within a year. UV1 is a patented, proprietary technology owned by Ultimovacs.

Published on : 09th October, 2023