Therapy wins rare pediatric disease designation for hard-to-treat glioma
FDA status awarded to iopofosine I 131 for young patients with brain cancer
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to iopofosine I 131 — now being tested in young people in a clinical trial in North America — as a potential treatment for inoperable relapsed or refractory (hard-to-treat) pediatric high-grade glioma (r/r pHGG).
The FDA awards this status to experimental therapies that have the potential to improve care for rare conditions that affect children. Its goal is to incentivize companies to invest in medications for rare pediatric diseases. The designation means that if iopofosine I 131 is ultimately approved, its developer Cellectar Biosciences will be given a voucher that can be used to get faster FDA review of a future application. Cellectar would be able to use this voucher or sell it to another company.
“Receiving rare pediatric disease designation for iopofosine I 131 underscores its potential to address one of the most devastating cancers affecting children and young adults,” James Caruso, president and CEO of Cellectar, said in a company press release.
Gliomas are cancers that occur due to the uncontrolled growth of cells in the brain. In children with r/r pHGG, the cancer grows rapidly and does not respond well to available treatments. According to Cellectar, most children with this type of cancer do not live longer than six months, and the median time before the disease progresses is usually less than three months.
Iopofosine I 131 is an investigational radiotherapeutic medicine, a small molecule designed to kill cancer cells by delivering them a radioactive compound. Caruso said that the new FDA designation “validates the promise of our targeted radiotherapeutic approach,” which the company is developing as a novel treatment for glioma and various other forms of cancer.
Clinicial trial now testing iopofosine I 131 in young people with r/r pHGG
Cellectar is sponsoring an ongoing Phase 1b clinical trial called CLOVER-2 (NCT05610891) to test iopofosine I 131 in young people with r/r pHGG. The trial, underway at seven sites in the U.S. and one in Canada, enrolled approximately 50 patients, ages 10-25.
Interim findings recently reported from six patients showed that the average time to disease progression was 5.4 months and the average overall survival time was 8.6 months, with some patients still alive at the time of the analysis. According to Caruso, this indicates “meaningful improvements in progression-free and overall survival.”
We believe iopofosine I 131 represents a compelling opportunity for strategic collaboration to accelerate development and bring a potentially first-in-class therapy to patients who urgently need new options.
Among the three patients who underwent additional treatment cycles — receiving at least four infusions of iopofosine — the average progression-free survival was 8.1 months, and the average overall survival was 11.5 months.
“We believe iopofosine I 131 represents a compelling opportunity for strategic collaboration to accelerate development and bring a potentially first-in-class therapy to patients who urgently need new options,” Caruso said.
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