Neurotech International (NTI) has flagged to its shareholders that the FDA hasn’t granted its product NTI164 orphan drug status.

Orphan drug status is that classification awarded to drugs by the FDA for, often, rare diseases. Companies can access tax credits for running trials, see less fees overall, and in some cases, the FDA will award market exclusivity for seven years.

However, on Friday, Neurotech confirmed the FDA has failed to deem the company’s target conditions – Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute- Onset Neuropsychiatric Syndrome (PANS) respectively – as being rare enough.

PANDAS is, for its part, what it sounds like – often referring to the development of obsessive compulsive tic symptoms following an infection of strep throat. The condition is regarded as an autoimmune issue.

The US-based National Institute of Mental Health (NIH) writes on its webpage for the condition that certain natural antibodies a human being produces to battle off strep can ‘malfunction’ and enter the brain where they can cause OCD symptoms – or worsen that underlying condition.

The FDA did not challenge that the condition exists, by any means. But it did take issue with the second condition, PANS.

All in all, the US-based medicines regulator isn’t convinced there’s enough evidence to suggest that PANS is a standalone condition compared to PANDA – which means the company doesn’t have two rare conditions to treat, just the one.

And when it comes to establishing what is rare and what isn’t, the FDA is further unsure Neurotech’s data is the most reliable when it points to prevalence rates. NTI points to a 1-in-200 prevalence in children across the USA, but on Friday, the FDA effectively asked for a please explain.

Neurotech now has 12 months to address a reply to the regulator and provide supporting evidence. The company wrote on Friday it is, in layman’s terms, making up its mind on what to do.

NTI last traded at 5.8cps.

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