The US Food and Drug Administration (FDA) has knocked back an application by Neurotech International seeking orphan drug designation for its broad-spectrum cannabinoid therapy aimed at children with neurological disorders.
Neurotech said the FDA considers the two conditions for which it was seeking orphan drug designation (ODD) – Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANDAS/PANS) – as conditions that “may not constitute the definition of a rare disease in the US”.
The company has a 12-month window to respond to the FDA’s concerns.
Despite the setback, the ASX-listed company said its PANDAS/PANS program remains “unaffected”, pointing to the strength of its clinical and safety data.
“The company notes the FDA had no objections to Neurotech’s submitted non-clinical and clinical evidence supporting the scientific rationale for the ODD,” it said.
The FDA cited two primary reasons for its decision. The regulator questioned whether PANS could be clearly distinguished as a standalone disease, and it also disagreed with the data Neurotech submitted to support the condition’s classification as “rare”.
Beyond PANDAS/PANS, Neurotech’s low-THC oral drug NTI164 – which contains CBDA, CBC, CBN, CBDP and CBDB – has been through clinical trials for Autism and Rett Syndrome.
A separate ODD application for Rett Syndrome is still under FDA review.
Orphan drug designation carries several advantages, including tax credits for clinical trial costs and the possibility of seven years of market exclusivity following approval.
