• Neurotech files for orphan drug designation for oral cannabinoid drug NTI164
  • Submission focused on rare paediatric disease
  • FDA expected to respond within three months


Special Report: Paediatric biopharmaceutical company Neurotech has filed for orphan drug designation (ODD) with the US Food and Drug Administration.

The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition.

ODD qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees and a potential seven years of market exclusivity after approval.

Neurotech (ASX:NTI) is seeking the designation for the use of its NTI164 treatment in children and adults diagnosed with Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS).

A response from the FDA Office of Orphan Products Development is anticipated within three months of filing.


A rare neurological disorder

The Orphan Drug Act defines a rare disease as a disease or condition that affects less than 200,000 people in the United States.

PANDAS/PANS is a rare neurological disorder predominately in children characterised by an infection triggered autoimmune response and associated neuroinflammation which results in a sudden, dramatic change in personality, displayed as obsessive-compulsive disorder (OCD), anxiety, tics or other abnormal movements and personality changes.

There are no approved therapies for PANDAS/PANS, globally – and the company estimates the annual market for the disorder is worth US$1.2 billion.


Promising clinical trials

Neurotech’s broad-spectrum oral cannabinoid drug therapy called NTI164 has already completed a Phase II/III randomised, double-blind, placebo-controlled clinical trial in Autism Spectrum Disorder (ASD).

Clinically meaningful and statistically significant benefits were reported across a number of clinically validated measures and excellent safety, the company says.

In addition, Neurotech has completed and reported statistically significant and clinically meaningful Phase I/II trials in ASD and PANDAS and PANS, along with Rett Syndrome. And Neurotech has received human ethics committee clearance for a Phase I/II clinical trial in spastic cerebral palsy.


This article was developed in collaboration with Neurotech, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.