Plus Therapeutics Granted U.S. FDA Orphan Drug Designation to REYOBIQ™ in Pediatric Malignant Gliomas

Plus Therapeutics, Inc. a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, today announced that the U.S. Food and that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to REYOBIQ (rhenium Re186 obisbemeda) for the treatment of pediatric malignant gliomas, are rare, aggressive brain tumors with poor outcomes, where current treatments often fail to prevent recurrence. It also includes the broader scope of progressive pediatric ependymoma.  

Pediatric malignant gliomas are rare, aggressive brain tumors with limited treatment options and poor outcomes, where current standards of care include surgery and radiation, which often fail to prevent recurrence. 

According to Towards Healthcare, the Orphan Oncology Market is projected to experience significant growth, with estimates suggesting the market size will increase from USD 16.55 trillion in 2026 to approximately USD 69.85 trillion by 2035, representing a compound annual growth rate (CAGR) of 17.35% from 2026 to 2035. Growth is driven by the rise in the number of rare diseases, and certain rare cancers are driving the demand for orphan drugs, with advancements leading to more effective treatments for rare diseases. 

About Leptomeningeal Metastases 

LM is a rare complication of cancer in which the primary cancer spreads to the cerebrospinal fluid (CSF) and leptomeninges surrounding the brain and spinal cord. All malignancies originating from solid tumors, primary brain tumors have this LM complication potential, with breast cancer as the most common cancer linked to LM, with 3-5% of breast cancer patients developing LM. 

It is noticed that lung cancer, GI cancers, and melanoma can also spread to the CSF and result in LM. 

About Plus Therapeutics 

Its Headquartered in Houston, Texas, It target radiotherapeutics for difficult-to-treat cancers of the central nervous system with the potential to enhance clinical outcomes. Combining image-guided local beta radiation and targeted drug delivery approaches, the Company is advancing a pipeline of product candidates with lead programs in leptomeningeal metastases (LM) and recurrent glioblastoma (GBM).  

It built a supply chain through strategic partnerships that enable the development, manufacturing, and future potential commercialisation of its products. 

A recent report by Towards Healthcare highlights that the Orphan Oncology Market is witnessing growth due to demand for regulatory incentives, technological advancements, and unmet clinical needs. It also reflects sustained demand fueled by rising cancer prevalence and improved diagnostic capabilities. 

North America, particularly the United States, is leading the market with a substantial number of orphan drug approvals and a strong regulatory framework that supports patient advocacy and research. In the Asia Pacific, the market is also growing, with increasing government programs and regulations that encourage orphan drug research and development.