Neuren Pharmaceuticals (ASX: NEU) has achieved another key regulatory milestone, receiving Rare Pediatric Disease Designation from the US FDA for its investigational drug NNZ-2591 in Pitt Hopkins syndrome (PTHS) and Angelman syndrome (AS). This adds to the company’s existing Rare Pediatric Disease Designation for Phelan-McDermid syndrome (PMS).
Potential for a Priority Review Voucher
With these designations in place, Neuren stands to gain a Priority Review Voucher (PRV) if NNZ-2591 secures FDA approval, provided the PRV program is reauthorized by Congress. PRVs are highly valuable, as they can fast-track regulatory review for another drug or be sold—with past vouchers fetching upwards of US$150 million. Neuren’s partner, Acadia Pharmaceuticals, previously sold a PRV granted for its Rett syndrome drug, DAYBUE™.
Neuren’s Expanding Neurology Pipeline
Neuren is focused on developing treatments for serious childhood neurological disorders that currently have few or no approved therapies. The company’s lead drug, DAYBUE™ (trofinetide), is already FDA-approved for Rett syndrome and is licensed globally to Acadia Pharmaceuticals. Meanwhile, NNZ-2591 continues to show promise, with positive Phase 2 trial results in PTHS, AS, and PMS.
This latest regulatory win strengthens Neuren’s position in rare disease drug development, potentially paving the way for faster approvals and strategic commercial opportunities.
