FDA Grants Rare Pediatric Disease Designation to ONC201 for the Treatment of H3 K27M-Mutant Glioma

Rare Daily Staff

The U.S. Food and Drug Administration granted Oncoceutics’ experimental therapy ONC201 Rare Pediatric Disease designation for rare pediatric brain tumors with a specific mutation known as H3 K27M-mutant gliomas.

ONC201 is a highly selective antagonist of dopamine receptor D2 (DRD2) and ClpP agonist that is able to penetrate the blood-brain-barrier. Oncoceutics has completed phase 1 studies with ONC201 in solid tumors and has additional phase 1/2 and phase 2 clinical programs in both solid and hematological malignancies ongoing.

The FDA previously granted Orphan Drug and Fast Track designations for ONC201 in H3 K27M-mutant gliomas.

“ONC201 has the potential to become the first FDA-approved therapy for patients with H3 K27M-mutant gliomas,” said Lee Schalop, CEO 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.”

The Rare Pediatric Disease Priority Review Voucher program 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.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Photo: Lee Schalop, CEO of Oncoceutics

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.