The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to the experimental radiation therapy rhenium Re186 obisbemeda — slated to be marketed under the brand name Reyobiq — as a potential treatment for pediatric malignant gliomas, a category of aggressive brain cancers that affect children.
The FDA grants this status to investigational medicines intended to treat conditions that affect fewer than 200,000 individuals in the U.S. The designation offers extra incentives to companies that are investing in potential therapies for rare diseases.
Among the perks, drugs granted this designation receive guaranteed seven years of market exclusivity, free from generic competitors, if the therapy is ultimately approved.
Plus Therapeutics, rhenium Re186 obisbemeda’s developer, plans to market the therapy under the name Reyobiq, according to a company press release announcing the new FDA status. The company noted that “the FDA granted orphan designation for malignant glioma more broadly than originally requested,” to include ependymoma, a rare type of brain tumor also affecting children.
The company has launched a Phase 1 study (NCT07061626) that aims to explore the safety of Reyobiq in children with high-grade gliomas or hard-to-treat ependymomas, which are tumors that arise from the membranes around the brain and/or spinal cord. The trial has not yet begun recruitment.
“Receiving orphan drug designation for Reyobiq in pediatric malignant gliomas, including the broader scope for progressive pediatric ependymoma, is an important milestone and further validates our approach to delivering targeted radiotherapy directly to [brain and spinal cord] tumors,” said Marc Hedrick, MD, president and CEO of Plus Therapeutics.
Radiation therapy being developed for a range of hard-to-treat cancers
Gliomas are nervous system cancers caused by the uncontrolled growth of glia, brain cells that normally help protect and support nerve cells. Radiation therapy can kill glioma cells with high potency, but it’s hard to get powerful radiation through the skull or spine and into the central nervous system to target a tumor without also damaging the surrounding tissue.
Plus’ therapy uses a small molecule carrying a radioactive payload that is delivered directly to the tumor via a surgical procedure. The goal is to administer radiation without having to shoot a beam through someone’s head, allowing a larger amount of cancer-killing radiation to be given to the tumor without causing substantial damage to nearby healthy parts of the brain.
Reyobiq is being developed as a potential therapy for a variety of hard-to-treat forms of cancer.
“We believe Reyobiq’s ability to deliver high-dose radiation precisely to tumor sites while minimizing exposure to healthy brain tissue has the potential to meaningfully improve outcomes in this underserved patient population,” Hedrick said. “This orphan designation reinforces the potential applicability of Reyobiq across a wider range of [brain and spinal cord] tumor indications and our continued advancement of Reyobiq across multiple [brain and spinal cord] cancer indications.”
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