The US Food and Drug Administration (FDA) has rejected an application from Neurotech International to grant orphan drug status for its broad-spectrum cannabinoid treatment for children with neurological conditions.
Neurotech said the FDA believes 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) – “may not constitute the definition of a rare disease in the US”.
Neurotech has 12 months to address the FDA’s concerns.
Despite the disappointing development, the ASX-listed firm said its plans for PANDAS/PANS remain “unaffected” given the strength of the 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.
Two main factors were cited by the FDA for its decision. It felt it was unclear a distinction could be made for PANS as a separate disease, while it did not agree with data provided by Neurotech to suggest it could be considered “rare”.
In addition to PANDAS/PANS, Neurotech’s low-THC oral drug NTI164, which contains CBDA, CBC, CBN, CBDP and CBDB, has undergone clinical trials for the treatment of Autism and Rett Syndrome.
An application for Rett Syndrome to receive orphan drug status is under review by the FDA.
Among the benefits of ODD are tax credits for clinical trials and the potential for seven years of market exclusivity after approval.
