FDA Grants Orphan Drug Status to Cintredekin Besudotox for Glioblastoma

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational therapy, cintredekin besudotox, for glioblastoma multiforme, according to a news release from Precision NeuroMed.

"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," Dr. Sandeep Kunwar, CEO and co-founder, Precision NeuroMed, said in the news release. "By combining innovative drugs with our next-generation delivery system, we hope to dramatically improve outcomes where few effective options currently exist."

The FDA grants orphan drug designation to medicines that aim to treat rare diseases, which affect fewer than 200,000 people in the U.S. This status helps make developing these treatments easier by offering benefits such as lower fees, research tax credits and seven years of exclusive approval once the drug is on the market.

Cintredekin besudotox, also known as IL13-PE38QQR, is a targeted protein therapy designed to kill tumor cells that express the IL-13 alpha-2 receptor (IL13aR), which is commonly found in glioblastoma. By focusing on this receptor, the treatment aims to destroy cancer cells while sparing healthy brain tissue.

Even though doctors better understand the genetics of glioblastoma and have found new drug targets, treating it remains difficult because the blood-brain barrier blocks many medicines from reaching the tumor. Precision NeuroMed’s approach uses convection-enhanced delivery, a technique that delivers treatment directly into the brain, allowing higher, more targeted doses of nanoparticles such as proteins, liposomes or gene therapies to reach cancer cells.

Each year, more than 12,000 people in the U.S. die from glioblastoma, and the average life expectancy after diagnosis is only 12 to 18 months, as per the release. Standard treatment includes surgery to remove as much of the tumor as possible, followed by radiation and chemotherapy with temozolomide — an approach that hasn’t changed since 2005. PNM is working on a more personalized treatment that targets the tumor’s unique features and location in the brain to improve how drugs reach cancer cells, with the goal of helping patients live longer and feel better.

Glioblastoma is a fast-growing brain cancer that starts in the glial, or supportive, tissue of the brain or spinal cord, as per the National Cancer Institute’s website. It’s the most common and aggressive type of brain tumor in adults and rarely affects the spinal cord. It’s also known as GBM or grade 4 astrocytoma.

Previous Orphan Drug Designation for Glioblastoma

In September, the FDA granted orphan drug designation to BA-101, an investigational therapy for patients with glioblastoma, according to a news release from NeuroNOS, a subsidiary of Beyond Air that manufactures the drug.

“Glioblastoma is one of the most common and deadliest brain cancers in adults, however, patients have seen little improvement in treatment options over the past several decades. Emerging industry research shows that [nitric oxide] is an important modulator of biological therapy response in glioblastoma,” said Amir Avniel, CEO of NeuroNOS, in the release. “We believe this data and the urgent unmet medical need have highlighted the opportunity for our groundbreaking science to develop small molecule therapies that balance nitric oxide levels in the brain. We believe [a nitric oxide] inhibition strategy has the potential to transform outcomes for patients.”

In a study published earlier this year in Genomic Press: Brain Medicine, researchers found that combining BA-101 with temozolomide, a standard brain cancer drug, reduced tumor size and improved survival in mice with temozolomide-sensitive glioblastoma cells.

Reference

1. “Precision NeuroMed Granted FDA Orphan Drug Designation by U.S. FDA for Treatment of Glioblastoma Multiforme (GBM).” Precision NeuroMed. Oct. 7, 2025.

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