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FDA grants orphan drug designation to VAL-083 for ovarian cancer
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The FDA granted orphan drug designation to dianhydrogalactitol for the treatment of patients with ovarian cancer who have failed or are unlikely to respond to modern chemotherapy regimens.
Dianhydrogalactitol (VAL-083, DelMar Pharmaceuticals) — a small-molecule chemotherapeutic — previously received orphan designation for the treatment of glioma and medulloblastoma.
“We are pleased to receive the designation, which is timely in light of new data presented this week with supporting the potential for dianhydrogalactitol in the treatment of ovarian cancer,” Jeffrey Bacha, chairman and CEO of DelMar Pharmaceuticals, said in a company-issued press release. “This announcement is representative of the progress we’ve made in developing VAL-083, which we believe positions the therapy as a viable treatment option for ovarian cancer patients.”
Results of more than 40 phase 1 and phase 2 studies suggested dianhydrogalactitol as a single agent or in combination with other therapies has clinical activity in a variety of malignancies, including brain, cervical, lung and ovarian cancers, as well as leukemia.
Preclinical data show the agent’s mechanism of action is distinct from the platinum-based chemotherapies commonly used for ovarian cancer treatment. Dianhydrogalactitol demonstrated the ability to circumvent cisplatin resistance in all ovarian cell lines tested, according to the press release.
Most patients with ovarian cancer who undergo first-line treatment with platinum-based chemotherapy respond; however, the majority relapse within 18 months. Prior studies suggest median survival among those with platinum-resistant disease is between 6 and 9 months.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — seven years of market exclusivity.