Octapharma Unveils Phase 3 Study Results for Innovative Treatment of Pediatric PANS

Octapharma Unveils Promising Phase 3 Study Results for PANZYGA in Treating Pediatric PANS

Octapharma has announced the results of its recent Phase 3 superiority study on PANZYGA®, an intravenous immune globulin treatment, showcasing its potential in treating Pediatric Acute-onset Neuropsychiatric Syndrome (PANS). This groundbreaking study involved a prospective, randomized, double-blind, placebo-controlled design aimed at comparing the effectiveness of PANZYGA® against a placebo in pediatric patients with moderate to severe PANS.

Understanding Pediatric Acute-onset Neuropsychiatric Syndrome (PANS)

PANS is a complex syndrome characterized by a sudden onset of obsessive-compulsive symptoms along with additional cognitive, behavioral, and neurological issues. This condition can lead to significant distress for affected children and their families, often requiring urgent medical attention to alleviate the debilitating symptoms. The trial aimed to highlight PANZYGA®'s therapeutic potential in mitigating these symptoms, thus providing hope for many families struggling with the condition.

Study Design and Key Findings

Conducted at various sites, the study enrolled 71 patients aged between 6 to 17 years. They were randomly assigned to receive either PANZYGA® or a placebo over a period of nine weeks, with the primary endpoint being the reduction in the severity of symptoms as measured by the Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS). Results indicated a substantial improvement of 31.1% in the PANZYGA® group compared to only 12.1% in the placebo group by the ninth week. This difference underscores the treatment's potential efficacy, although it fell just short of statistical significance with a p-value of 0.072.

In addition to the primary endpoint, secondary endpoints like the Clinical Global Impression Improvement (CGI-I) also provided encouraging data. The CGI-I score improvements were statistically significant, indicating that patients treated with PANZYGA® experienced a notable enhancement in their overall condition, including behavioral and cognitive symptoms, with a p-value of 0.017.

The study also employed a crossover analysis following the initial nine weeks, allowing participants from both groups to switch the treatment they received. This method enabled researchers to further investigate the sustainability of symptom relief. The continued improvement in CY-BOCS scores among those initially treated with PANZYGA® highlights its enduring impact even after the treatment period.

Implications for Future Care

Principal Investigator Dr. Michael Daines emphasized that while the primary outcome did not meet its predefined success, the significant improvement observed in the CGI-I reflects PANZYGA®'s potential in managing the complex web of issues associated with PANS. “These outcomes suggest that PANZYGA® might address the wider disease burden associated with pediatric neuropsychiatric conditions, presenting an essential avenue for families navigating these challenging situations,” Dr. Daines elaborated.

Acknowledgements and Future Steps

The PACE Foundation, along with various advocacy groups, played a pivotal role in supporting the recruitment process for this essential trial. Paul Ryan, Co-Founder and President of the PACE Foundation, conveyed gratitude to Octapharma for their dedication to families affected by this intricate disease.

As treatment for PANS can be particularly challenging due to its often sudden onset and complex symptomatology, Octapharma is dedicated to continuing research in this area to confirm and expand on the findings of this trial, aiming to develop effective therapeutic strategies for these vulnerable patients.

About PANZYGA®

PANZYGA® is an immune globulin intravenous (human) preparation known for treating primary humoral immunodeficiency, chronic immune thrombocytopenia in adults, and chronic inflammatory demyelinating polyneuropathy. As a treatment strategy, PANZYGA® stands out for its safety profile and efficacy, offering hope to patients requiring immune support.

Octapharma remains at the forefront of human protein manufacturing, with the overarching goal of enhancing patient care and improving life quality through innovative therapies worldwide. With a robust network and advanced production capabilities, the company signifies a commitment to continuous improvement and exploration in the medical field.