The FDA and European Medicines Agency granted orphan drug designation to zotiraciclib for the treatment of glioma, according to the agent’s manufacturer.
Zotiraciclib (Adastra Pharmaceuticals) — a multikinase inhibitor — is being evaluated in two phase 1b clinical studies of patients with glioblastoma, the most common glioma histology.
A multiarm dose-finding study sponsored by NCI is designed to assess zotiraciclib plus temozolomide for recurrent malignant gliomas.
A three-parallel cohort, nonrandomized multicenter study conducted by European Organisation for Research and Treatment of Cancer (EORTC) is evaluating zotiraciclib alone, in combination with radiation or in combination with temozolomide for elderly patients with IDH1 R132H-nonmutant and MGMT promoter-unmethylated anaplastic astrocytoma or glioblastoma.
“We are very pleased to have been granted orphan drug designation by the FDA and European Medicines Agency for zotiraciclib in the treatment of glioma,” Scott Megaffin, CEO of Adastra, said in the press release. “These designations are important in the continued development of zotiraciclib and the ongoing clinical trials in the U.S. at the NCI and throughout Europe with the EORTC.
“Though rare, glioblastoma is one of the most devasting and difficult-to-treat cancers, urgently necessitating the development of new treatments with unique mechanisms of action,” Megaffin added. “Data from the ongoing clinical trials of zotiraciclib have been encouraging and are suggestive of a therapy that is safe and capable of eliciting clinical benefit in high-grade gliomas. We greatly look forward to reporting topline data from the NCI phase 1b study in early 2020, as well as completing enrollment in two of the EORTC study cohorts later in the year.”
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 — 7 years of market exclusivity.