While the data from the IVIG trial is under review, we are pleased that Octapharma has officially released a statement regarding the IVIG trial. Below is an excerpt from their announcement: 

“From baseline (week 0) to week 9, there was a mean improvement of 31.1% (SD 40.68) in the PANZYGA® group CY-BOCS score as compared to 12.1% (SD 68.39) in the placebo group. […] While the mean ratio of the total CY-BOCS score at week 9 improved in the PANZYGA® group as compared to the placebo group (primary endpoint), the ratio difference between the two groups was not statistically significant […] The research team also observed that the difference in the improvement of the Clinical Global Impression (CGI-I) score at week 9 (secondary endpoint) was statistically significant […] 

The study utilized a crossover analysis from week 9 to week 18 where the PANZYGA® group participants received the placebo and the participants who had received the placebo until week 9 were given PANZYGA® through week 18 […] The CY-BOCS score improvement was more prominent in the participant group that initially received PANZYGA® until week 9, which researchers attributed to a continued response to the PANZYGA® treatment in the first half of the study.”

Both measurements of results (primary and secondary endpoints) showed a positive treatment effect for participants receiving PANZYGA® and notably, the secondary endpoint achieved statistical significance—an important milestone. While the primary endpoint did not reach what is considered statistical significance, we’re encouraged by the trial’s overall results.

In Octapharma’s statement, Dr. Michael Daines, Principal Investigator and Division Chief of Pediatric Allergy and Immunology at the University of Arizona, shared:

“While this Phase III trial did not meet its primary efficacy outcome of OCD symptom improvement [...] it revealed promising insights: the key secondary endpoint of Clinical Global Impression (CGI) showed statistically significant and clinically relevant improvement. [...] CGI reflects the holistic impact of PANS/PANDAS on a patient’s life, encompassing behavioral, cognitive, and physical symptoms. [...] These results suggest that PANZYGA® may address the broader disease burden [...] offering a potential therapeutic pathway for families navigating this complex condition. Further research into this investigational use of PANZYGA® is needed to confirm this suggestion.”

Octapharma USA President Flemming Nielsen added:

“PANS is a very challenging disease for the medical community and families. Parents are confronted with the challenge of seeing their children afflicted with sudden-onset, debilitating, and difficult to treat OCD as well as other behavioral and cognitive issues. Octapharma looks forward to bringing relief to children and their families in the near future.”

Please note, that the review process may take additional time before any decisions about the IVIG trial are finalized. PACE will continue to provide updates via our website, newsletter, and social media as new developments emerge.

We are deeply proud and grateful for our fellow advocacy organizations whose dedication helped raise awareness of this trial among families affected by PANS/PANDAS. Additionally, we’d like to acknowledge the principal investigators and multitude of dedicated medical professionals who contributed to the IVIG trial. Finally, we’d like to express sincere appreciation to Octapharma for sponsoring this global study and for their compassion and continued commitment to these families. Regardless of the final regulatory outcome, this trial represents meaningful progress in supporting children and families in need.

You can read the full statement from Octapharma here.

With gratitude,
The PACE Team