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# Glioblastoma Multiforme Drug Gets Orphan Status
- URL: https://www.fdaweb.com/glioblastoma-multiforme-drug-gets-orphan-status/
- Published: 2025-10-10T12:00:00.000Z
- Updated: 2026-09-14T15:23:52.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5160025

FDA has granted Precision NeuroMed an orphan drug designation for IL13-PE38QQR (cintredekin besudotox), an investigational therapy targeting glioblastoma multiforme, an aggressive and often lethal form of brain cancer. The cytotoxic protein is engineered to attack cells expressing the IL-13 alpha-2 receptor — a marker commonly found on glioblastoma cells but largely absent in normal brain tissue, the company says. The selectivity of the target is designed to minimize collateral damage to healthy brain cells while delivering potent anti-tumor activity.

Precision NeuroMed says that to overcome the long-standing challenge of the blood–brain barrier, it employs its convection-enhanced delivery platform, which allows direct infusion of therapeutic agents — including proteins, nanoparticles, and gene therapies — into brain tissue at concentrations that would be difficult to achieve via systemic administration.

Glioblastoma affects roughly 12,000 people in the U.S. each year, with a median survival time of just 12 to 18 months following diagnosis and a five-year survival rate of about 5%, according to the company. It says the current standard of care — surgical resection followed by radiation and temozolomide — has seen little innovation since 2005.