News|Articles|October 8, 2025
Cintredekin Besudotox Earns FDA Orphan Drug Designation in Glioblastoma
Author(s)Sabrina Serani
Fact checked by: Andrea Eleazar, MHS
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Key Takeaways
- Cintredekin besudotox targets IL-12 alpha-2 receptor-expressing tumor cells, using convection-enhanced delivery to bypass the blood-brain barrier for glioblastoma treatment.
- Orphan drug designation offers tax credits, FDA fee exemptions, and 7 years of market exclusivity, benefiting Precision NeuroMed.
FDA grants orphan drug designation to cintredekin besudotox, offering new hope for glioblastoma treatment with targeted delivery methods.
The FDA has granted orphan drug designation to cintredekin besudotox (IL13PE38) for the treatment of glioblastoma.1
Cintredekin besudotox is a potent cytotoxic protein designed to target and kill cells expressing the IL-12 alpha-2 receptor (IL13aR), which is uniquely present on tumor cells including glioblastoma. The drug delivery platform uses convection-enhanced delivery to bypass the blood-brain barrier and enable direct, targeted administration of the therapy.
"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 fo-founder of Precision NeuroMed, in a press release. "By combining innovative drugs with our next-generation delivery system, we hope to dramatically improve outcomes where few effective options currently exist."
Orphan drug designation is given to therapies intended to treat, diagnose, or prevent rare diseases that affect fewer than 200,000 patients in the US. With this designation, Precision NeuroMed, the sponsor, is eligible for tax credits for clinical research, exemption from FDA fees, and 7 years of market exclusivity upon approval.2
The 5-year survival rate for glioblastoma is 5%, with an average life expectancy after diagnosis of 12 to 18 months. Standard therapy comprises maximal tumor resection and radiation therapy with temozolomide, which was approved in 2005.1 This designation offers hope for a novel, effective therapy for this patient population.
REFERENCES:
1. Precision NeuroMed Granted FDA Orphan Drug Designation by U.S. FDA for Treatment of Glioblastoma Multiforme (GBM). News release. Precision NeuroMed. October 7, 2025. Accessed October 7, 2025. https://tinyurl.com/ff92uuwu
2. Designating an Orphan Product: Drugs and Biological Products. US FDA. Updated August 12, 2024. Accessed October 7, 2025. https://tinyurl.com/5ckfaxtv
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