Octapharma Announces Phase 3 Study Results for Pediatric Acute-onset Neuropsychiatric Syndrome

Octapharma recently unveiled findings from a significant phase 3 study aimed at evaluating the efficacy of PANZYGA® for treating pediatric acute-onset neuropsychiatric syndrome (PANS). This large-scale trial was designed as a prospective, randomized, double-blind, parallel group, crossover, placebo-controlled study. The primary focus was to compare the performance of PANZYGA®, an immune globulin intravenous, human - ifas 10% liquid preparation, against a placebo in mitigating symptoms associated with PANS.

The research utilized the Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) to measure improvements at the nine-week mark, which served as the primary endpoint. Additional analysis was conducted using secondary endpoints, particularly the Clinical Global Impression (CGI-I), at the same intervals of week 9 and week 18.

From the study's commencement to the ninth week, participants treated with PANZYGA® demonstrated a mean improvement of 31.1% on the CY-BOCS score, compared to a 12.1% improvement observed in the placebo group. However, the statistical analysis of the primary endpoint did not reveal a significant difference between the two groups, indicated by a p-value of 0.072. Despite this, the secondary endpoint revealed a statistically significant improvement in CGI-I scores for PANZYGA® recipients, with a p-value of 0.017.

The study's design included a crossover phase between weeks 9 and 18, where initial PANZYGA® recipients were switched to the placebo, and those initially on the placebo received PANZYGA®. Continual improvement in the CY-BOCS scores was noted in both groups, with the initial PANZYGA® cohort showing more pronounced benefits, suggesting a sustained response to the treatment.

Dr. Michael Daines, the study's principal investigator and Division Chief of Pediatric Allergy and Immunology at the University of Arizona College of Medicine, highlighted the significance of the secondary endpoint findings. He noted that while the primary objective wasn't fully met, the CGI-I results are encouraging as they reflect broader improvements in the behavioral, cognitive, and physical symptoms experienced by PANS patients. Dr. Daines emphasized the potential of PANZYGA® to offer a new therapeutic option for managing PANS and called for further research to substantiate these findings.

The PACE Foundation played a crucial role in supporting the trial's recruitment efforts. Paul Ryan, Co-Founder and President of the foundation, expressed gratitude towards Octapharma's commitment to addressing the challenges faced by families affected by PANS. Flemming Nielsen, President of Octapharma USA, acknowledged the significant impact PANS has on families, emphasizing the company's dedication to alleviating the difficulties faced by children with sudden and severe neuropsychiatric symptoms.

The trial enrolled 71 children aged 6 to 17 years, all diagnosed with moderate to severe PANS. Participants underwent three infusions of either PANZYGA® or a placebo over two days every three weeks across a nine-week period. This was followed by a double-blind, crossover safety and efficacy phase with similar treatment administration.

PANZYGA® is primarily used for treating primary humoral immunodeficiency, chronic immune thrombocytopenia, and chronic inflammatory demyelinating polyneuropathy. Despite its therapeutic potential, PANZYGA® carries warnings for thrombosis and renal dysfunction risks, necessitating careful monitoring and management during treatment.

Octapharma, headquartered in Switzerland, is recognized as a leading producer of human proteins, with products distributed in 118 countries and operations supported by advanced R&D facilities and numerous plasma donation centers. The company continues to focus on innovative treatments across immunotherapy, hematology, and critical care, bolstered by decades of experience in patient care.