Neuren Pharmaceuticals (ASX: NEU) has announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for NNZ-2591, its investigational treatment for Pitt Hopkins syndrome (PTHS). This designation is intended to accelerate the development and regulatory review of promising treatments for serious conditions with no approved therapies.
Neuren’s Phase 2 clinical trial of NNZ-2591 in children with Pitt Hopkins syndrome showed 82% of participants experienced improvement in key areas, including communication, social interaction, cognition, and motor abilities.
What Fast Track means for NNZ-2591
The FDA’s Fast Track designation allows Neuren to:
- Hold more frequent meetings with the FDA to discuss trial design and development.
- Receive priority review or accelerated approval if eligibility criteria are met.
- Submit parts of its New Drug Application (NDA) on a rolling basis instead of waiting for full completion.
About Pitt Hopkins syndrome
Pitt Hopkins syndrome is a rare neurodevelopmental disorder caused by mutations in the TCF4 gene on chromosome 18. It is estimated to affect between 1 in 34,000 and 1 in 41,000 people. Symptoms include severe intellectual disability, speech impairment, seizures, and breathing abnormalities. There are currently no FDA-approved treatments for the condition.
Neuren’s broader pipeline
NNZ-2591 is also in development for other neurodevelopmental disorders, including Phelan-McDermid syndrome and Angelman syndrome. The company previously secured FDA approval for DAYBUE™ (trofinetide), a treatment for Rett syndrome, which is now licensed to Acadia Pharmaceuticals.
Neuren CEO Jon Pilcher said the Fast Track designation reflects the urgent unmet need in Pitt Hopkins syndrome and strengthens the company’s pathway to regulatory approval.
