FDA Grants Rare Pediatric Disease Designation to ONC201 for the Treatment of H3 K27M-Mutant Glioma
Philadelphia, PA (July 21, 2020) – Oncoceutics, a clinical-stage drug discovery and development company, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to Oncoceutics’ investigational cancer drug ONC201 for the treatment of “H3 K27M-mutant glioma”.
The Rare Pediatric Disease Priority Review Voucher program, which was created as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, is intended to incentivize the development of new therapies for rare pediatric diseases. Should ONC201 be approved by the FDA for H3 K27M-mutant glioma, the Rare Pediatric Disease Designation may enable Oncoceutics to receive a Rare Pediatric Disease Priority Review Voucher. A priority review voucher, if granted, would provide Oncoceutics with the ability to receive a priority review of a subsequent marketing application for a different product, and this opportunity for a priority review may be sold or transferred.
“ONC201 has the potential to become the first FDA-approved therapy for patients with H3 K27M-mutant gliomas,“ said Lee Schalop, MD, Chief Executive Officer of Oncoceutics. “The FDA’s recognition of the high prevalence and dismal prognosis of the H3 K27M mutation in pediatric patients is a significant milestone for the development of ONC201.”
“This Rare Pediatric Disease Designation is in addition to the Orphan Drug and Fast Track designations for ONC201 in H3 K27M-mutant gliomas that we previously received and builds upon our presentation to the public advisory committee meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee in June of last year,” said Martin Stogniew, Chief Development Officer of Oncoceutics. "The eligibility to receive a priority review voucher from the FDA provides significant potential upside as we continue to work diligently towards the completion of our pivotal program in H3 K27M-mutant gliomas.”
Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds called imipridones that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist and ClpP agonist. The company is supported by grants from NCI, FDA, The Musella Foundation, and the National Brain Tumor Society, and a series of private and public partnerships. Visit Oncoceutics.com for more information.