Neurotech chases another orphan drug designation

• Neurotech files orphan drug designation for oral cannabinoid drug NTI164 to treat Rett Syndrome
• It’s the second indication Neurotech has filed an orphan drug designation for after PANDAS/PANS
• Neurotech expects a response from US FDA on its application within three months of filing

Special Report: Neurotech has submitted a request with the US Food and Drug Administration for orphan drug designation (ODD) for use of its broad-spectrum cannabinoid drug NTI164 in children and adults diagnosed with Rett Syndrome. 

Clinical-stage biopharmaceutical focused predominantly on paediatric neurological disorders Neurotech International (ASX:NTI) has filed its second ODD with the FDA within two months for NTI164.

NTI said the FDA has authority to grant ODD 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.

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

Rare disorder affecting girls

Rett Syndrome is a rare genetic neurological and developmental disorder and is almost exclusively the result of a mutation or mutations in the methyl CpG binding protein 2 (MECP2) gene located on the X chromosome, which is required for normal brain development and function.

The syndrome occurs almost exclusively in girls, with an incidence of one in 10,000 female live births with the prevalence of ~15,000 girls and women in the US and 350,000 globally.

NTI recently reported positive results from the extension phase of Phase 1/2 trial of NTI164, with all 14 female paediatric patients showing significant clinical improvement at 20 weeks.

The company said a response from the FDA Office of Orphan Products Development is anticipated within 90 days of filing or in late November 2024.

ODD application for PANDAS/PANS

In July NTI filed its first ODD with the FDA for use of NTI164 to treat Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS).

PANDAS/PANS is a rare neurological disorder predominantly 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, 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$1.2bn.

Based on estimated timelines a response from FDA on its ODD application for PANDAS/PANS is anticipated within a couple of months.

This article was developed in collaboration with Neurotech International, 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.