The US FDA has granted fast-track designation for Neuren's NNZ-2591 for the treatment of Pitt Hopkins syndrome (PTHS).
The designation is designed to facilitate the development and expedite the review of drugs to treat severe conditions.
It provides for more frequent engagement between the company and the FDA to discuss the candidate's development plan and ensure the collection of appropriate data needed to support its approval. It also makes the candidate eligible for Accelerated Approval and Priority Review.
Currently, no treatments have been approved to treat PTHS.
PTHS is a neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18. The incidence of PTHS has been estimated at between one in 34,000 and one in 41,000 people.
Characteristics of PTHS are a range of developmental delays with moderate-to-severe intellectual disability and behavioural differences, hyperventilation and/or breath-holding while awake, seizures, gastrointestinal issues, lack of speech, sleep disturbance, stereotypic hand movements and distinctive facial features.
In Neuren’s Phase 2 clinical trial of NNZ-2591 in children with PTHS, 82 per cent of participants showed improvement, including communication, social interaction, cognition and motor abilities.