Latest Rett Syndrome News

Neuren Pharmaceuticals has dosed the first patient in its groundbreaking Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome, a rare genetic disorder with no approved therapies. The trial marks a significant step forward for the PMS community and rare disease drug development.

The US Congress has reauthorized the Rare Pediatric Disease Priority Review Voucher program through September 2029, a move that could significantly benefit Neuren Pharmaceuticals as it advances its rare disease drug candidates.

Acadia Pharmaceuticals has received a negative vote from the European Medicines Agency’s committee on its application to market trofinetide for Rett syndrome in the EU, prompting plans for a re-examination request.

Neurotech International has secured a Rare Pediatric Disease Designation from the FDA for its lead drug NTI164 targeting Rett syndrome, alongside publishing promising clinical data and raising $8.7 million to fuel further development.

Neuren Pharmaceuticals’ partner Acadia projects DAYBUE sales to reach around US$700 million by 2028, driven by US market expansion and international approvals. The launch of a new formulation and ongoing clinical trials in Japan underpin this optimistic outlook.

Neuren Pharmaceuticals has published promising Phase 2 trial results for NNZ-2591 in Phelan-McDermid syndrome, underpinning its ongoing Phase 3 Koala trial recruiting in the US.

Neuren Pharmaceuticals has secured Health Canada approval to expand its pivotal Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome into Canadian sites, complementing ongoing US enrolment.

Neurotech International has locked in $4 million through a share placement to advance its promising paediatric neurological drug, NTI164. The raise, supported by both new and existing investors including company directors, aims to fund critical clinical and regulatory milestones.

Neuren Pharmaceuticals’ partner Acadia secures FDA approval for DAYBUE STIX, a new powder formulation of trofinetide offering greater flexibility for Rett syndrome patients. This milestone expands treatment options and sets the stage for increased royalty revenue in 2026.

Neurotech International has received a $4.73 million R&D tax incentive refund, reinforcing its clinical development efforts in paediatric neurological disorders. The funds will support ongoing trials and working capital needs.

Neuren Pharmaceuticals reported a milestone quarter with DAYBUE net sales reaching US$101.1 million, driving a 24% jump in royalty income. The expanding patient base and community physician uptake signal strong growth ahead.

Neurotech International has initiated an Authorised Prescriber program for its lead therapy NTI164, expanding access for children with neurodevelopmental disorders in Australia. The company also secured a Rare Pediatric Disease Designation from the US FDA for NTI164 in Rett Syndrome, marking a key regulatory milestone.