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The regulatory agency has granted orphan drug designation to cintredekin besudotox for the treatment of patients with glioblastoma.
The FDA has granted orphan drug designation to cintredekin besudotox (IL13-PE38QQR) for the treatment of patients with glioblastoma.1
“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,” Sandeep Kunwar, MD, chief executive officer and cofounder of Precision NeuroMed, stated in a 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 blood-brain barrier can reduce the efficacy of systemic therapies delivered through the bloodstream. Cintredekin besudotox is a potent cytotoxic protein intended to selectively kill IL-13 alpha-2 receptor–expressing cells, sparing damage to normal brain tissue. The agent uses convection-enhanced delivery (CED) to bypass the blood-brain barrier, resulting in direct, targeted administration of nanoparticles, including proteins, liposomes, and gene therapy at therapeutic concentrations to the brain.
CED of cintredekin besudotox was compared with carmustine chemotherapy in adult patients recurrent with glioblastoma multiforme in the phase 3 PRECISE trial (NCT00076986).2 Patients were randomly assigned 2:1 to receive cintredekin besudotox or carmustine. cintredekin besudotox was administered at a dose of 0.5 µg/mL for a total flow rate of 0.75 mL/h over 96 hours via 2 to 4 intraparenchymal catheters placed after tumor resection. Carmustine was administered at a dose of 7.7 mg per wafer for a maximum of 8 wafers immediately following tumor resection.
The primary end point was overall survival (OS). Secondary and tertiary end points included safety and health-related quality-of-life assessments.
Between March 2004 and December 2005, 296 patients were enrolled at 52 centers. Demographic and baseline characteristics were balanced between the 2 treatment arms.
Results from the trial indicated that the median OS was 36.4 weeks (95% CI, 34.14-45.57) with cintredekin besudotox vs 35.3 weeks (95% CI, 29.86-47.29) with carmustine in the intention-to-treat population (P = .476). In the efficacy evaluable population, the median OS was 45.3 weeks (95% CI, 34.71-52.57) for cintredekin besudotox and 39.8 weeks (95% CI, 34.86-50.43) for carmustine (P = .310).
With respect to safety, the adverse effect profile was similar in both arms, apart from pulmonary embolism, which occurred at a higher frequency in the cintredekin besudotox arm, at 8% vs 1%, respectively (P = .014).
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