Plus Therapeutics Secures FDA Orphan Drug Designation For REYOBIQ In Pediatric Brain Tumours
Plus Therapeutics gains FDA Orphan Drug Designation for REYOBIQ in pediatric malignant gliomas and ependymoma, advancing targeted radiotherapy for rare brain tumors.
Breaking News
Apr 09, 2026
Vaibhavi M.
Plus Therapeutics, Inc. has announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) to REYOBIQ™ (rhenium Re186 obisbemeda) for the treatment of pediatric malignant gliomas. These rare and aggressive brain tumors have limited treatment options and poor outcomes, with current therapies often failing to prevent recurrence.
The designation was granted more broadly than initially requested, extending to additional conditions such as pediatric ependymoma. Orphan Drug Designation is intended to support the development of treatments for rare diseases affecting fewer than 200,000 people in the United States and offers benefits such as market exclusivity, tax incentives, and reduced regulatory fees.
“Receiving orphan drug designation for REYOBIQ in pediatric malignant gliomas, including the broader scope for progressive pediatric ependymoma, is an important milestone and further validates our approach to delivering targeted radiotherapy directly to CNS tumors,” said Marc Hedrick, M.D., President and Chief Executive Officer of Plus Therapeutics. “We believe REYOBIQ’s ability to deliver high-dose radiation precisely to tumor sites while minimizing exposure to healthy brain tissue has the potential to meaningfully improve outcomes in this underserved patient population. This orphan designation reinforces the potential applicability of REYOBIQ across a wider range of CNS tumor indications and our continued advancement of REYOBIQ across multiple CNS cancer indications.”
This milestone builds on recent progress for REYOBIQ, including regulatory engagement with the FDA and encouraging clinical data from the ReSPECT-LM trial. The company is actively advancing the therapy through ongoing Phase 1 and Phase 2 studies, focusing on its potential in central nervous system cancers.
Additionally, Plus Therapeutics has received clearance for its Investigational New Drug (IND) application to study REYOBIQ in pediatric patients with high-grade gliomas and ependymoma. These developments collectively strengthen the company’s efforts to address critical unmet needs in pediatric oncology.
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