Is NNZ-2591 legal in the US?
Not yet; it is still in trials.
CAnimal and cell studiesIn clinical trialsas of 2026-10-27
Not approved by the FDA for any use. NNZ-2591 is an investigational oral drug from Neuren Pharmaceuticals being tested in rare neurodevelopmental disorders, and the published human data are from an open-label phase 2 trial in Phelan-McDermid syndrome. A randomized, placebo-controlled phase 3 trial in children with Phelan-McDermid syndrome started in November 2025 and is recruiting.
More on NNZ-2591
Citations
- Neumeyer 2025 (published online December 2025; Feb 2026 issue), Neurology Genetics: NNZ-2591 in children and adolescents with Phelan-McDermid syndrome, single-group open-label phase 2 trial doi.org
- ClinicalTrials.gov NCT05025241: an open-label study of oral NNZ-2591 in Phelan-McDermid syndrome (PMS-001) clinicaltrials.gov
- BioWorld 2024: Neuren's NNZ-2591 meets phase 2 endpoints in Pitt Hopkins syndrome (company report) bioworld.com
- ClinicalTrials.gov: NCT07281079, phase 3 trial of NNZ-2591 in Phelan-McDermid syndrome clinicaltrials.gov
Reference information only, not medical advice. Drawn from the NNZ-2591 article, fact-checked against the cited sources. No dosing anywhere on this site.