Neuren Pharmaceuticals recieves FDA designation
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The news: Neuren Pharmaceuticals has received a Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for its drug to treat Phelan-McDermid syndrome.
The context: Neuren's drug NNZ-2591 to treat the debilitating childhood neurodevelopmental disorder is toward the end of its phase 2 meeting with the FDA to discuss the development program for the drug.
The designation from the FDA may allow the company to qualify for a priority review that aims to incentivise drug development for serious rare pediatric diseases.
Neuren said that during its phase 2 clinical trial of NNZ-2591 clinicians and caregivers across multiple efficacy measures saw significant improvement in patients.
The source: ASX announcement
