On October 7, 2025 Precision NeuroMed (PNM), a clinical-stage biotechnology company pioneering advanced drug delivery technologies for central nervous system (CNS) diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its investigational therapy for glioblastoma (GBM) (Press release, Precision NeuroMed, OCT 7, 2025, View Source [SID1234656496]).
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PNM is reintroducing cintredekin besudotox (IL13-PE38QQR), a potent cytotoxic protein designed to target and kill cells that express the IL-13 alpha-2 receptor (IL13aR), uniquely present on tumor cells such as glioblastoma. The protein’s selective expression allows it to precisely attack cancer cells while minimizing damage to normal brain tissue.
Despite advancements in understanding the genetics of glioblastoma and identifying novel drug targets, the blood–brain barrier (BBB) remains a significant obstacle, severely limiting the effectiveness of many potential therapies delivered through the bloodstream. PNM’s platform uses convection-enhanced delivery (CED) to bypass the BBB and enable direct, targeted administration of nanoparticles, including proteins, liposomes, and gene therapy at therapeutic concentrations to the brain.
"Receiving Orphan Drug Designation is an important milestone for Precision NeuroMed as we advance our mission to transform treatment for patients with glioblastoma, one of the most aggressive and devastating brain cancers," said Sandeep Kunwar, MD, CEO and Co-Founder, Precision NeuroMed. "By combining innovative drugs with our next-generation delivery system, we hope to dramatically improve outcomes where few effective options currently exist."
Each year, more than 12,000 individuals in the United States are expected to succumb to glioblastoma. The five-year survival rate for patients is just 5%, with an average life expectancy of 12 to 18 months following diagnosis. Standard treatment consists of maximal tumor resection followed by radiation therapy with temozolomide with no major innovations since the approval of temozolomide for glioblastoma in 2005.
PNM is developing a personalized approach that targets both molecular and regional aspects of tumor cells within the brain. By improving drug delivery at the site of disease, PNM aims to extend survival and enhance quality of life for patients with glioblastoma in a meaningful way.
Orphan Drug Designation from the U.S. Food and Drug Administration is reserved for therapies intended to treat, diagnose, or prevent rare diseases affecting fewer than 200,000 people in the United States. This designation provides development benefits — such as tax credits for eligible clinical research and exemption from applicable FDA user fees — and, upon approval for the designated indication, confers seven years of market exclusivity.