Neurotech chasing prized US orphan drug designation

Paediatric biotech Neurotech has filed for orphan drug designation with the US FDA

Neurotech is focused predominately on paediatric neurological disorders. Pic: via Getty Images.

• 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.

Originally published as Neurotech chasing prized US orphan drug designation