The European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to niraparib for the treatment of glioma, including glioblastoma (GBM), following the U.S. FDA’s grant of the same status in November 2025. Glioma is an umbrella term that includes glioblastoma, the target indication. This designation is supported by the Ivy Brain Tumor Center’s Hybrid Phase 0/2 study. The results and opinion were based on the fulfillment of key ODD criteria, including evidence supporting the potential benefit of niraparib in newly diagnosed glioblastoma and the disease's serious, life-threatening nature.
The EMA grants orphan designation to investigational drugs for the treatment, prevention, or diagnosis of rare, life-threatening, or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU, or for which the drug would otherwise be commercially non-viable. Where satisfactory treatment methods are already authorized, the drug must demonstrate the potential to offer a significant benefit over those existing options to patients. While ODD is an important step in the research process, niraparib has not yet been approved to treat GBM, and its safety and effectiveness for this use are still being studied.
“Niraparib receiving ODD in Europe is a testament to the rigor of the Ivy Center team: the trial design, the protocol execution, the pharmacokinetic and pharmacodynamic analyses, the tissue processing, and the clinical care behind every enrolled patient,” says Dr. Nader Sanai, director of the Ivy Brain Tumor Center at Barrow Neurological Institute and study chair. “We aim to redefine central nervous system (CNS) oncology for patients, families, and caregivers. It brings hope during the most difficult moments of their lives.”
The Gliofocus Study is a global, randomized, controlled Phase 3 clinical trial comparing niraparib vs temozolomide in patients with newly-diagnosed, MGMT-unmethylated GBM. The global Phase 3 Gliofocus study completed enrollment in May 2026. Patients and care partners can learn more about Gliofocus by visiting Gliofocus.org and NCT06388733 .
The Ivy Center is home to the largest Phase 0 drug development program for brain cancer worldwide. Click here for a complete list of Ivy Center clinical trials.