Neuren Pharmaceuticals (ASX:NEU) has received Fast Track designation from the US Food and Drug Administration (FDA) for its investigational therapy NNZ-2591 for the treatment of Phelan-McDermid syndrome (PMS), a rare neurodevelopmental disorder with no approved therapies.
The designation, designed to speed the development and review of medicines addressing serious unmet needs, coincides with Phelan-McDermid Syndrome Awareness Day on 22 October. NNZ-2591 is currently being evaluated in Koala, the first-ever Phase 3 randomised, double-blind, placebo-controlled trial for PMS in children aged 3 to 12 years. Neuren said it reached alignment with the FDA on the single Phase 3 trial design and endpoints needed to support a New Drug Application.
“This milestone represents an important step for patients and their families,” said CEO Jon Pilcher. “Fast Track designation gives us a clear regulatory pathway, with opportunities to accelerate review and bring a much-needed treatment to a community that currently has none. We applaud the work of the Phelan-McDermid Syndrome Foundation and CureSHANK for their leadership in raising awareness and supporting research.”
PMS is caused by a deletion or mutation in the 22q13 region of chromosome 22, affecting the SHANK3 gene. It is estimated to affect between 1 in 8,000 and 1 in 15,000 people, with symptoms including developmental and intellectual impairment, delayed or absent speech, autism traits, low muscle tone, motor delays, epilepsy, and behavioural and socialisation difficulties.
The FDA’s Fast Track process provides more frequent engagement with regulators, eligibility for accelerated approval and rolling review, and priority consideration once an application is submitted. NNZ-2591 has also received Fast Track designation for Angelman syndrome and Pitt Hopkins syndrome, highlighting its potential across multiple neurodevelopmental conditions.