MVdeltaC Earns Orphan Drug Status for Pleural Mesothelioma

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MVdeltaC for the treatment of patients with pleural mesothelioma, a rare and aggressive cancer with high unmet medical need and limited therapeutic options, according to a news release from Oncovita, the developer of the drug.

“Receiving orphan drug designation from the FDA for MVdeltaC marks a major milestone for Oncovita and validates our approach of harnessing the potential of measles vaccine viruses to treat solid tumors, particularly rare and devastating cancers such as pleural mesothelioma,” said Stéphane Altaba, CEO of Oncovita, in the news release. “This regulatory support strengthens our strategy to advance innovative immunotherapies as we prepare to enter clinical development with MVdeltaC by 2026.”

The FDA grants orphan drug designation to encourage therapies for rare diseases that affect fewer than 200,000 people annually in the U.S. This designation grants developers perks including waived fees for FDA applications, as many as seven years of market exclusivity following approval, and tax credits toward qualifying clinical trials.

“With this designation, Oncovita is now well-positioned to enter the U.S. market with its modified attenuated measles virus for the treatment of pleural mesothelioma. This recognition highlights the promise of this novel approach against one of the most aggressive cancers in medicine,” Dr. Stéphane Champiat, head of medical affairs at Oncovita, added in the news release.

Champiat is also an associate professor for the Department of Investigational Cancer Therapeuticsat The University of Texas MD Anderson Cancer Center, Houston, Texas

MVdeltaC is an experimental immunotherapy that uses a weakened, modified measles virus designed to target and destroy tumor cells while also activating the immune system. With this treatment, Oncovita aims to improve outcomes in advanced solid tumors such as pleural mesothelioma by combining direct cancer cell destruction with immune stimulation.

Pleural mesothelioma is a rare, aggressive cancer of the lung lining, most often caused by long-term asbestos exposure. About 3,000 people are diagnosed annually in the U.S. Prognosis is generally poor, especially when the disease recurs or stops responding to treatment, and options remain limited.

Previous Findings

MVdeltaC showed strong immune-stimulating and tumor-killing activity in preclinical studies, according to a previous news release from Oncovita published in 2023. Based on these findings, a first-in-human trial is planned for patients with solid tumors, with particular interest in those whose disease no longer responds to checkpoint inhibitors.

Researchers created a modified version of the measles virus called MVdeltaC by removing one of its genes linked to illness. They tested MVdeltaC in lab studies on more than 40 types of human cancer cells, including mesothelioma, lung, bladder, ovarian, cervical and liver cancers. The vaccine was also tested in several animal models, including mice implanted with human tumors and mice with intact immune systems. Scientists studied how the virus works using immune cells taken from people.

In preclinical studies, MVdeltaC showed strong anti-tumor activity both in vitro and in vivo. It was two to three times more potent than standard measles virus in killing human tumor cell lines and was active in over 70% of those tested. In mice with human mesothelioma, a single low dose significantly reduced tumor size, and in patient-derived xenograft models, weekly MVdeltaC dosing controlled tumor growth in mesothelioma and bladder cancer.

In immunocompetent mice, three intratumoral injections led to tumor regression or complete disappearance in 70% of animals, with long-term immune protection shown by resistance to tumor re-challenge. Ex vivo experiments confirmed MVdeltaC triggered a strong immune response, including activation of dendritic cells, phagocytosis of tumor cells and presentation of tumor antigens to T cells. The therapy also produced high levels of defective interfering genomes, which may drive its immune-stimulating effects through the RIG-I pathway.

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