FDA Grants Rare Pediatric Designation to Treatment With NEO100 in Brain Cancer

The FDA grants Rare Pediatric Disease Designation for NEO100 in the treatment of pediatric-type diffuse high-grade gliomas.

The United States Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for NEO100 (perillyl alcohol) in the treatment of pediatric-type diffuse high-grade gliomas, a life-threatening brain cancer, according to a news release from NeOnc Technologies Holdings, Inc., in turn, expediting potential approval of the therapeutic agent.

This decision is based on evidence that the disease primarily affects individuals from birth to 18 years and qualifies as a rare disease. Moreover, the regulatory agency has granted the designation under Section 529(a)(3) of the Federal Food, Drug and Cosmetic Act (FD&C Act) to acknowledge the urgent need for the treatment of patients with pediatric-type diffuse high-grade gliomas.

“This designation marks a significant milestone in our efforts to develop innovative therapies for children battling this aggressive form of brain cancer,” Amir Heshmatpour, Executive Chairman of NeOnc Technologies Holdings, Inc., stated in the news release “We remain committed to advancing NEO100 through clinical development to bring new hope to patients and families facing this devastating disease.”

“Currently, there is a dynamic market for PRV’s, which are quite valuable,” Heshmatpour added. “This potential asset further underscores the value of our progress, especially for the patients and families we aim to serve.”

With this Rare Pediatric Disease Designation, NeOnc Technologies may be eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of NEO100’s marketing application. The PRV program incentivizes the development of new treatments for rare pediatric diseases by offering an expedited regulatory review process.

“Receiving the Rare Pediatric Disease Designation for NEO100 is a crucial step forward in our mission to develop effective treatments for children facing diffuse high-grade gliomas,” said Dr. Thomas Chen, CEO and chief science officer of NeOnc Technologies Holdings, Inc., in the news release. “This designation not only validates the potential of our research but also strengthens our commitment to delivering innovative therapies that can make a real difference in children’s lives.”

To conclude the news release, the company shares that they are continuing to progress NEO100 through clinical trials and that they remain dedicated to working closely with the FDA, as well as any other regulatory agency, in order to treat patients in need.

More Information on the Future Investigation of NEO100

Currently, a phase 1b trial is set to evaluate treatment with NEO100 for the treatment of patients with pediatric-type diffuse high-grade gliomas. The trial has not yet begun the recruitment of patients.

This clinical trial is designed to evaluate the safety and effectiveness of NEO100, as well as determine the optimal dose of the agent. The study will assess how well the drug is delivered to the brain and the pharmacokinetics of the agent (how the body interacts with the administered agent for the entire duration of exposure). Patients will receive NEO100 through the nose in a controlled dose-escalation approach to determine the safest and most effective dosage. NEO100 is a non-sterile solution for intranasal administration, and in other words, is given as a nasal spray rather than an injection or pill.

This method is being studied based on previous research conducted on treatment with intranasal drug delivery, as well as research with NEO100; previous research suggests that delivering medication through the nose may allow it to reach the brain more efficiently while reducing side effects in the rest of the body. 

The trial is open to patients with certain types of aggressive pediatric brain tumors, including newly diagnosed and recurrent diffuse midline glioma, diffuse hemispheric glioma, high-grade gliomas and other malignant brainstem or posterior fossa tumors.

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