The Children's Tumor Foundation
07/22/2026 | Press release | Distributed by Public on 07/23/2026 08:15
Trineumin Receives FDA Rare Pediatric Disease Designation for NF2
PRG S&Tech has announced that its investigational therapy, Trineumin (PRG-N-01), has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for the treatment of NF2-related schwannomatosis (NF2-SWN).
The designation is intended to encourage the development of therapies for serious rare diseases that primarily affect children. It follows several other regulatory milestones for Trineumin, including FDA Orphan Drug Designation, FDA Fast Track designation, and Orphan Medicinal Product Designation from the European Medicines Agency (EMA).
Trineumin is an investigational oral therapy designed to target a signaling pathway disrupted by mutations in the NF2 gene. According to the company, the drug works by regulating the interaction between the proteins TβR1 and RKIP, with the goal of slowing or preventing tumor growth.
The therapy is currently being evaluated in a Phase 1/2a clinical trial in South Korea. The Phase 1 portion has completed dosing and safety evaluation, and the study is progressing toward determining the recommended Phase 2 dose.
While Trineumin remains an investigational therapy and has not yet been approved for use, the FDA's Rare Pediatric Disease Designation represents another important step in its clinical development and highlights the ongoing effort to expand treatment options for people living with NF2-SWN.
Watch CTF's NF Knowledge Series webinar and learn more about this clinical trial here.
CTF's role: accelerating promising options for the NF2-SWN community
This news reflects what CTF's mission looks like in action: advancing strong science so potential therapies can reach patients faster. CTF is supporting this trial, including funding for clinical MRI, which is central to assessing whether a therapy affects tumor growth over time. We are grateful to the NF2-SWN community-patients, families, clinicians, and researchers whose participation and partnership make progress possible.
The Children's Tumor Foundation published this content on July 22, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on July 23, 2026 at 14:15 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]