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Niraparib Granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for the Treatment of Glioma
• With over 300,000 new cases annually, and a 5-year survival rate of less than 5%, GBM is both the most common and lethal brain cancer in adults.
• 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.
The EMA has granted ODD to niraparib for the treatment of glioma, including 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.
“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 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.”
Nader Sanai, MD
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.
About Niraparib
Niraparib, is an oral, once-daily PARP inhibitor with current indications in first-line and recurrent maintenance treatment for advanced ovarian cancer. It is currently approved under the brand name Zejula for the treatment of ovarian cancer.
About Gliofocus
Gliofocus is a multi-center, open-label, randomized study launched in June 2024 following encouraging results from early-phase trials conducted at the Ivy Center. In collaboration with GSK, the Gliofocus study enrolled 455 participants across more than 90 clinical sites in 11 countries. This Phase 3 Gliofocus study, sponsored by the Ivy Brain Tumor Center with funding and collaboration from GSK, compares niraparib monotherapy to the standard of care, temozolomide and radiotherapy, in adult patients with newly diagnosed GBM tumors that lack a key chemical modification, called MGMT unmethylated.
About Ivy Brain Tumor Center
Ivy Brain Tumor Center at Barrow Neurological Institute in Phoenix, AZ, is a tertiary care and nonprofit translational research program that employs bold, early-phase clinical trial strategies to identify new treatments for aggressive brain tumors, including GBM. Our leading experts in neurosurgical oncology, neuro-oncology, radiation oncology, neuroradiology, neuropathology and neuroscience nursing treat more patients annually than any other brain tumor center in the United States. The Ivy Center’s Phase 0 clinical trials program is the largest in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. In addition, unlike conventional clinical trials focusing on single drugs, the Ivy Center’s accelerated program tests therapeutic combinations matched to individual patients. We leave no stone unturned in the pursuit of hope and healing. Learn more at IvyBrainTumorCenter.org. Follow the Ivy Brain Tumor Center on Facebook, Instagram, X, and LinkedIn.