Neuren Pharmaceuticals (ASX:NEU) Receives FDA Rare Pediatric Disease Designation
Regulatory Designations
Neuren Pharmaceuticals has received Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for NNZ-2591 in Pitt Hopkins syndrome (PTHS) and Angelman syndrome (AS). The company had previously obtained the designation for NNZ-2591 in Phelan-McDermid syndrome (PMS).
Future Implications
With designations in PMS, PTHS, and AS, Neuren may be awarded a priority review voucher if the Rare Pediatric Disease PRV program is reauthorised by the US Congress and NNZ-2591 receives marketing authorisation for any of these indications. Previously, Neuren’s partner Acadia Pharmaceuticals received a PRV for DAYBUE™ (trofinetide) in Rett syndrome and sold the PRV for US$150 million.
Motley Fool contributor Abbie Stokes has no position in any of the stocks mentioned. The Motley Fool Australia's parent company Motley Fool Holdings Inc. has no position in any of the stocks mentioned. The Motley Fool Australia has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. This article contains general investment advice only (under AFSL 400691). Authorised by Scott Phillips.
This article was generated using GPT-4o mini, a Large Language Model (LLM), to generate summaries of investing news. While AI is generating the content, we know better than to blindly trust our future robot overlords, and every article is edited and fact-checked by an editor holding the appropriate credentials. The Motley Fool Australia stands behind the work of our editorial team and takes ultimate responsibility for the content of everything published by The Capital Club.