Neurotech Secures Rare Pediatric Disease Designation for NTI164 in Rett Syndrome

Neurotech International (ASX: NTI) has notched another major regulatory milestone in its mission to treat rare neurological disorders. The United States Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for the company’s lead drug candidate, NTI164, for the treatment of Rett syndrome — a rare and debilitating neurodevelopmental condition affecting mostly girls.

This new designation adds to the Orphan Drug Designation already held by NTI164 for the same indication and significantly strengthens Neurotech’s regulatory and commercial position in the US market. The RPDD program is designed to encourage development of treatments for diseases affecting fewer than 200,000 people in the United States, and it comes with valuable benefits for drug developers.

These include enhanced regulatory guidance during development, potential priority review, exemption from certain FDA fees, and a key commercial edge — seven years of US market exclusivity if the treatment receives approval.

CEO Dr Anthony Filippis said the FDA’s recognition was a major endorsement of NTI164’s therapeutic promise.

“The granting of Rare Pediatric Disease Designation by the FDA marks another major milestone for NTI164 and underscores its therapeutic potential for Rett syndrome,” said Dr Filippis. “Combined with the existing Orphan Drug Designation, this reinforces the significance of our program and our commitment to developing much-needed treatments for children and families affected by these challenging conditions.”

NTI164 is a proprietary cannabinoid therapy derived from a unique strain of Cannabis sativa, with both anti-inflammatory and neuroprotective properties. Neurotech has designed it specifically for paediatric neurological disorders, and it is currently being studied across several clinical programs.

In addition to Rett syndrome, NTI164 has demonstrated clinically meaningful and statistically significant benefits in trials for Autism Spectrum Disorder (ASD), Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS), and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS).

The company is also preparing to commence a Phase I/II trial in spastic cerebral palsy, marking a further extension of its platform into unmet neurological conditions.

The RPDD status could also become a financial asset in its own right. Companies that receive approval for a treatment with RPDD may be eligible to receive a Rare Pediatric Disease Priority Review Voucher, which can be used to expedite the review of another drug or sold to other pharmaceutical companies — often for significant sums.

With momentum building across its clinical pipeline and now dual regulatory designations in hand, Neurotech is strategically positioned to advance NTI164 through further clinical development and towards potential commercialisation in the world’s largest pharmaceutical market.