NEU.ASX: Fast Track granted by FDA for NNZ-2591 in Pitt Hopkins

Fast Track granted by FDA for NNZ-2591 in Pitt Hopkins

Stock	Neuren Pharmaceuticals Ltd (NEU.ASX)
Release Time	19 Feb 2025, 1 p.m.
Price Sensitive	Yes

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FDA Grants Fast Track for Neuren's NNZ-2591 in Pitt Hopkins Syndrome

Key Points

FDA grants Fast Track designation for NNZ-2591 to treat Pitt Hopkins syndrome
82% of participants showed improvement in Phase 2 trial for communication, social interaction, cognition and motor abilities
Fast Track aims to facilitate development and expedite review of drugs for serious conditions with unmet need

Full Summary

Neuren Pharmaceuticals (ASX: NEU) announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for NNZ-2591 for the treatment of Pitt Hopkins syndrome (PTHS). Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. In Neuren's Phase 2 clinical trial of NNZ-2591 in children with PTHS, 82% of participants showed improvement, including in communication, social interaction, cognition and motor abilities. PTHS is a rare neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene, characterized by developmental delay, intellectual disability, behavioral differences, seizures, and other symptoms. With no treatments currently approved for PTHS, the Fast Track designation aims to help get this important new drug to patients earlier.