FDA shocks Neurotech with rejection of orphan drug status for life-changing PANDAS/PANS treatment

Neurotech International Limited is facing an uphill battle after the United States Food and Drug Administration (FDA) declined the company’s request for orphan drug status for its promising drug, NTI164, intended to treat Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS). Despite Neurotech’s significant progress in clinical trials, the FDA’s refusal raises critical questions about the future of treatment options for these rare, debilitating disorders.

Neurotech, a biopharmaceutical development company focused on paediatric neurological disorders, announced that the FDA had not granted Orphan Drug Designation (ODD) for NTI164, citing concerns that PANDAS/PANS may not meet the criteria for rare disease classification in the United States, where a rare disease is defined as affecting fewer than 200,000 people. The FDA pointed to inconsistencies in the prevalence data, suggesting that PANDAS/PANS may be more common than initially believed.

However, Neurotech is not backing down. The company remains committed to its development plans, driven by over 52 weeks of clinical data demonstrating the safety and efficacy of NTI164 in reversing immune dysregulation in PANDAS/PANS patients. The FDA’s decision does not directly impact the ongoing clinical trials, and Neurotech has been granted a 12-month window to address the agency’s concerns and submit further supporting data.

FDA’s rationale challenges Neurotech’s approach

The FDA’s rejection of orphan drug status for NTI164 has sparked concern in the medical community, particularly among experts who recognize the urgent need for effective treatments for PANDAS/PANS, conditions that cause sudden onset of neuropsychiatric symptoms, such as obsessive-compulsive disorder (OCD), anxiety, and mood disturbances, typically triggered by infections. The PANDAS/PANS Research Consortium, in collaboration with the National Institute of Mental Health (NIMH), has worked to establish diagnostic criteria and treatment guidelines, yet the disorder remains under-recognized and under-treated.

One of the key factors in the FDA’s decision was its uncertainty over the classification of PANS as a distinct disease. The FDA has requested additional data from Neurotech to clarify the diagnosis, especially in light of evolving clinical standards. Neurotech plans to collaborate with global experts to strengthen the case for PANS being recognized in the World Health Organization’s International Classification of Diseases (ICD-11), potentially paving the way for future regulatory approvals.

Expert opinions suggest a path forward

Experts in the field, such as Professor Russell Dale, Co-Principal Investigator of Neurotech’s clinical trials, have stressed the significance of the company’s findings. The proteomic data from PANDAS/PANS children, published in 2024, provides critical insights into the disorder’s underlying mechanisms. Professor Dale believes that Neurotech’s work could shape the future of PANDAS/PANS treatment, particularly if further genomic analysis supports the current findings. His team is pushing for global recognition of the disorder, advocating for updated classifications and diagnostic criteria that would allow patients to access treatments like NTI164 more easily.

Looking ahead: Neurotech’s plans remain intact

Despite this regulatory setback, Neurotech’s development strategy remains unaffected. The company continues to focus on PANDAS/PANS while simultaneously exploring the potential of NTI164 in other neurological conditions, including Rett Syndrome. Neurotech is currently awaiting a decision from the FDA on an additional orphan drug request for NTI164 related to Rett Syndrome, expected before December 2024.

The FDA’s decision, though disappointing, has provided Neurotech with crucial regulatory insights. The company remains optimistic about the future of NTI164 and is determined to bring new therapies to market, especially for conditions with limited treatment options. With its clinical advisory group of global thought leaders and its strong clinical data, Neurotech is well-positioned to address the FDA’s concerns and advance the treatment of PANDAS/PANS.