The U.S. Meals and Drug Administration (FDA) has granted uncommon pediatric drug designation for iopofosine-131 in inoperable relapsed or refractory pediatric high-grade glioma (pHGG), in accordance with a information launch from Cellectar Biosciences.
The FDA’s uncommon pediatric illness designation program helps the event of therapies for critical situations that primarily have an effect on kids underneath 18.
“Receiving uncommon pediatric illness designation for iopofosine-131 underscores its potential to deal with probably the most devastating cancers affecting kids and younger adults. Mixed with the encouraging interim outcomes from our CLOVER-2 pHGG research, which confirmed significant enhancements in progression-free and general survival, this designation additional validates the promise of our focused radiotherapeutic strategy,” James Caruso, president and CEO of Cellectar, acknowledged within the information launch. “We consider iopofosine-131 represents a compelling alternative for strategic collaboration to speed up growth and convey a doubtlessly first-in-class remedy to sufferers who urgently want new choices.”
Interim findings from the CLOVER-2 research have been just lately shared on the American Affiliation for Most cancers Analysis Particular Convention on Pediatric Most cancers, held in September 2025. The section 1b trial is evaluating iopofosine-131, a focused radiotherapy, in kids, adolescents and younger adults in america and Canada with relapsed or refractory pediatric high-grade glioma, a uncommon and aggressive mind most cancers.
In an oral presentation in the course of the convention, the corporate’s chief working officer, Jarrod Longcor, reported encouraging early outcomes.
Amongst sufferers who acquired at the very least 55 millicuries (mCi) of the overall administered dose (6 sufferers), the typical progression-free survival (PFS) was 5.4 months and the typical general survival (OS) was 8.6 months, with each ongoing on the time of the report. All sufferers skilled illness management, which the Response Evaluation in Pediatric Neuro-Oncology committee notes can correlate with improved survival.
Three sufferers who acquired at the very least 4 infusions confirmed a median PFS of 8.1 months and an zos of 11.5 months, ongoing, with two experiencing measurable tumor shrinkage.
Particular person Outcomes Reported With Iopofosine-131 in Pediatric Mind Cancers
Two case research offered as a part of the CLOVER-2 trial replace highlighted encouraging outcomes in younger sufferers with uncommon and aggressive mind tumors handled with iopofosine-131.
Case Research 1: A 25-year-old man with diffuse hemispheric glioma carrying the H3 G34R/V mutation, who had undergone three earlier therapies, acquired 4 doses of iopofosine-131 totaling 126.6 mCi (40 mCi/m² per dose). Roughly eight months after screening, his goal lesion had decreased by greater than 50%. His progression-free survival was 10.9 months, and as of July 25, 2025, he remained alive greater than 18 months after beginning therapy.
Case Research 2: A 15-year-old lady with ependymoma, who had acquired eight prior therapies, was handled with 4 doses totaling 58.9 mCi of iopofosine I 131 (20 mCi/m² per dose). Her tumor dimension decreased from 252 mm² to about 141 mm². Her progression-free survival was 11.2 months, and as of July 22, 2025, she remained alive greater than 17 months after therapy initiation.
How Protected Is Iopofosine-131 for Youngsters and Younger Adults With Mind Most cancers?
Iopofosine-131 was typically nicely tolerated within the CLOVER-2 trial, with unintended effects in line with beforehand reported security findings. Notably, no sufferers skilled coronary heart, kidney or liver toxicity, and there have been no instances of peripheral neuropathy or important bleeding.
The therapy’s security profile mirrored its focused design, with minimal results outdoors the blood system. The most typical unintended effects have been hematologic, together with low platelet counts (thrombocytopenia), low white blood cell counts (neutropenia) and anemia, all of which have been anticipated and manageable. No treatment-related deaths have been reported.
How Does the CLOVER-2 Research Take a look at Iopofosine-131 in Pediatric Mind Most cancers?
The section 1b CLOVER-2 research is enrolling kids, adolescents and younger adults in america and Canada who’ve relapsed or refractory pediatric high-grade glioma. Its objective is to evaluate how protected and tolerable iopofosine-131 is at completely different dosing schedules.
One group receives two doses of 20 mCi/m², given 14 days aside for 2 cycles, with an elective third cycle. The opposite group receives three cycles of 10 mCi/m², additionally given 14 days aside, with an elective fourth cycle.
Researchers will consider how nicely the therapy controls tumor development by measuring PFS and OS, in addition to tumor quantity discount, to assist decide the optimum dose for later-phase research.
Reference
- “Cellectar Biosciences Receives Uncommon Pediatric Illness Designation from U.S. Meals and Drug Administration for Iopofosine I 131 in Relapsed or Refractory Pediatric Excessive-Grade Glioma.” Cellectar Biosciences. Information Launch. Oct. 27, 2025.
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