Octapharma Releases Results for Phase 3 Superiority Study of Pediatric Acute-onset Neuropsychiatric Syndrome

Clinical Global Impression Data Showed Statistically Significant & Clinically Relevant Improvement in Pediatric PANS Patients

PARAMUS, N.J., May 28, 2025 /PRNewswire/ -- Octapharma has released the results from its prospective, randomized, double-blind, parallel group, crossover, placebo-controlled phase 3 study comparing the effectiveness of PANZYGA^® (immune globulin intravenous, human - ifas) 10% Liquid Preparation versus placebo in patients with pediatric acute-onset neuropsychiatric syndrome (PANS).

The CY-BOCS score (Children's Yale-Brown Obsessive-Compulsive Scale) measured at 9 weeks was utilized as the primary endpoint for assessing PANZYGA^® effectiveness in reducing the severity of PANS symptoms. In addition, other secondary endpoints such as the Clinical Global Impression (CGI-I) were also measured. The endpoints were measured at week 9 and week 18.

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. Researchers compared the CY-BOCS score of the participants in the two groups as a ratio at week 9 and week 0 (CY-BOCS score at week 9/CY-BOCS score at week 0). 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 with a probability-value (p-value) of 0.072. 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 between both groups with a higher improvement in the IVIG group (p-value = 0.017). Please visit euclinicaltrials.eu for the study data.

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. Researchers observed the mean total CY-BOCS score in both treatment arms continued to improve. 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.

"While this Phase III trial did not meet its primary efficacy outcome of OCD symptom improvement measured by CY-BOCS, it revealed promising insights: the key secondary endpoint of Clinical Global Impression (CGI) showed statistically significant and clinically relevant improvement," said Principal Investigator Michael Daines, M.D., Division Chief of Pediatric Allergy and Immunology at the University of Arizona College of Medicine. "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 in pediatric acute-onset neuropsychiatric syndromes, 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."

The PACE Foundation, along with fellow advocacy groups, is proud to have supported recruitment for this important trial. "We're grateful to Octapharma for their compassion and ongoing commitment to families affected by this challenging disease," said Paul Ryan, PACE Foundation Co-Founder and President.

A Challenging Disease for Families & Providers
PANS is diagnosed in children who experience sudden dramatic, often overnight, onset of obsessive-compulsive symptoms and/or severe eating restrictions, along with at least two other cognitive, behavioral, or neurological symptoms. Brain inflammation can occur when the body's immune system mistakenly attacks healthy brain cells.

"PANS is a very challenging disease for the medical community and families," said Octapharma USA President Flemming Nielsen. "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."

Octapharma enrolled 71 patients from age 6 to 17 with a confirmed diagnosis of moderate to severe PANS in the prospective, randomized, double-blind, parallel group, placebo-controlled superiority study. (ClinicalTrials.gov Identifier: NCT04508530). Participants received three infusions of PANZYGA^® or placebo over two days every three weeks for a total of nine weeks, with an additional double-blind, crossover safety and efficacy follow-up phase of three infusions of PANZYGA^® or placebo administered over two days every three weeks for a total of nine weeks.

The primary objective of the study was to assess whether PANZYGA^® is superior to placebo (0.9% w/v sodium chloride) for reducing the severity of symptoms associated with PANS in pediatric patients. The secondary objectives were to evaluate the sustainability of the reduction of the severity of symptoms in pediatric patients treated with PANZYGA^®; and assess the efficacy of PANZYGA^® treatment in reducing functional impairment associated with PANS.

About PANZYGA^®
PANZYGA^® is an immune globulin intravenous, (human) - ifas 10% liquid preparation indicated for:
- Primary humoral immunodeficiency (PI) in patients 2 years of age and older;
- Chronic immune thrombocytopenia (ITP) in adults; and
- Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults.

WARNING: THROMBOSIS, RENAL DYSFUNCTION, and ACUTE RENAL FAILURE
– Thrombosis may occur with immune globulin intravenous (IGIV) products, including PANZYGA^®. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors.
– Renal dysfunction, acute renal failure, osmotic nephropathy, and death may occur with the administration of IGIV products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products containing sucrose. PANZYGA^® does not contain sucrose.
– For patients at risk of thrombosis, renal dysfunction, or renal failure, administer PANZYGA^® at the minimum infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.

Please see full prescribing information at hcp.panzygausa.com/pi.

About Octapharma
Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines.

Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care.

Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centers across Europe and the US. Octapharma has over 40 years of experience in patient care. The company's American subsidiary, Octapharma USA, is located in Paramus, N.J. For more information, please visit octapharmausa.com.

PANZ-0850

SOURCE Octapharma