Panzyga (immune globulin intravenous, human-ifas) / Octapharma, Pfizer - LARVOL DELTA

Empirical Time-Series Analysis (1991–2023) of Immune Thrombocytopenia Medications in U.S. Medicaid Programs (ISPOR 2025) - "Comparative market-share analyses were conducted across therapeutic classes to identify key drivers of market changes, including policy shifts, competition, and new drug entries The utilization trends revealed Rituxan dominated until 2020, after biosimilars (Ruxience, Truxima) gained traction, with Ruxience leading. In the IVIG category, Gammagard consistently held the highest utilization, peaking in 2022, followed by Rhophylac and Privigen. Among TPO-RAs, Promacta surpassed Nplate in 2018, reflecting a shift in prescribing patterns. Pricing data highlighted Promacta as the most expensive TPO-RA, Panzyga led IVIG pricing by 2019... This analysis highlights evolving patterns in the utilization, reimbursement, and pricing of ITP therapies within Medicaid programs, shaped by factors like the introduction of biosimilars, competitive market dynamics, and clinical advancements. These findings provide valuable insights for healthcare policymakers, payers, and clinicians to..."

Medicaid • Reimbursement • US reimbursement • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

IVIG in the Treatment of Autoimmune Small Fiber Neuropathy with TS-HDS, FGFR-3, or Plexin D1 Antibodies (clinicaltrials.gov) - P2 | N=20 | Active, not recruiting | Sponsor: Henry Ford Health System | Recruiting ➔ Active, not recruiting

Enrollment closed • Immunology • Pain

NGAM-13: Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS) (clinicaltrials.gov) - P3 | N=71 | Completed | Sponsor: Octapharma | Active, not recruiting ➔ Completed

Head-to-Head • Trial completion • Pediatrics • Psychiatry

PROSID: Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study) (clinicaltrials.gov) - P3 | N=240 | Active, not recruiting | Sponsor: Octapharma | Recruiting ➔ Active, not recruiting

Enrollment closed • Chronic Lymphocytic Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Oncology

Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P4 | N=6 | Terminated | Sponsor: Octapharma | N=20 ➔ 6 | Active, not recruiting ➔ Terminated | Trial primary completion date: Apr 2024 ➔ Sep 2023; FDA released Octapharma from the pediatric post marketing requirement.

Enrollment change • Trial primary completion date • Trial termination • Hematological Disorders • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

NGAM-13: Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS) (clinicaltrials.gov) - P3 | N=71 | Active, not recruiting | Sponsor: Octapharma | Trial completion date: Jun 2024 ➔ Oct 2024 | Trial primary completion date: Jun 2024 ➔ Oct 2024

Head-to-Head • Trial completion date • Trial primary completion date • Pediatrics • Psychiatry

NGAM-13: Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS) (clinicaltrials.gov) - P3 | N=70 | Active, not recruiting | Sponsor: Octapharma | Terminated ➔ Active, not recruiting

Enrollment closed • Head-to-Head • Pediatrics • Psychiatry

NGAM-13: Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (PANS/PANDAS) (clinicaltrials.gov) - P3 | N=70 | Terminated | Sponsor: Octapharma | Trial completion date: Oct 2024 ➔ May 2024 | Recruiting ➔ Terminated | Trial primary completion date: Oct 2024 ➔ May 2024; Medical Director decision; statistical considerations unrelated to safety

Head-to-Head • Trial completion date • Trial primary completion date • Trial termination • Pediatrics • Psychiatry

PROSID: Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study) (clinicaltrials.gov) - P3 | N=240 | Recruiting | Sponsor: Octapharma | Trial completion date: Oct 2024 ➔ Aug 2025 | Trial primary completion date: Oct 2024 ➔ Aug 2025

Trial completion date • Trial primary completion date • Chronic Lymphocytic Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Oncology

Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P4 | N=20 | Active, not recruiting | Sponsor: Octapharma | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS, FGFR-3, or Plexin D1 Antibodies (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: Henry Ford Health System | Not yet recruiting ➔ Recruiting

Enrollment open • Immunology • Pain

PROSID: Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study) (clinicaltrials.gov) - P3 | N=240 | Recruiting | Sponsor: Octapharma

Trial completion date • Trial primary completion date • Chronic Lymphocytic Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Oncology

IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS, FGFR-3, or Plexin D1 Antibodies (clinicaltrials.gov) - P2 | N=20 | Not yet recruiting | Sponsor: Henry Ford Health System

Trial completion date • Trial primary completion date • Immunology • Pain

NGAM-11: Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients (clinicaltrials.gov) - P3 | N=30 | Recruiting | Sponsor: Octapharma | Not yet recruiting ➔ Recruiting

Enrollment open • Pain • Pediatrics

An early onset of acute Inflammatory Demyelinating Polyneuropathy (AIDP) or Guillain-Barre Syndrome along with Covid-19. (EUSEM 2023) - "Treatment was substituted with intravenous immunoglobulin therapy (Panzyga 100 mg/ml, 150g in total)...It is important to mention that the patient had a Sars-cov-19 infection and this specific finding illustrates how there is a variety of infectious diseases that can lead to GBS. Plasmapheresis can cause an allergic reaction, but it is vital to attempt second line treatment, such as intravenous immunoglobulin therapy."

Allergy • Cardiovascular • Complement-mediated Rare Disorders • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Novel Coronavirus Disease • Pain • Respiratory Diseases

NGAM-11: Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients (clinicaltrials.gov) - P3 | N=30 | Not yet recruiting | Sponsor: Octapharma | Trial primary completion date: Dec 2025 ➔ Jun 2026

Trial primary completion date • Pain • Pediatrics

Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study. (PubMed, Drug Saf) - P3 | "Infusions of 10% IVIg at doses up to 2.0 g/kg with high infusion rates were safe and well tolerated in patients with CIDP."

Journal • Dermatitis • Dermatology • Immunology • Pain

Octapharma Puts a Spotlight on the Infection Burden in Patients With Chronic Lymphocytic Leukaemia (CLL) at the European Hematology Association (EHA) Hybrid Congress 2023 (Businesswire) - "Real-world data and expert perspectives on state-of-the-art clinical practice for the management of infection in patients with CLL will be showcased during the Octapharma Update-in-Hematology session at the EHA Hybrid Congress on June 9, 2023, in Frankfurt, Germany....Finally, Professor Livio Trentin, University of Padua, Italy, will speak about his experience using subcutaneous immunoglobulin therapy for infection prophylaxis in patients with CLL. In addition, he will share his first-hand experience with ongoing research into this area, including the PRO-SID clinical trial and its potential for primary infection prophylaxis. The session will round off with an exciting panel discussion bringing together the audience and experts to exchange views on challenging cases of infections associated with CLL."

Observational data • Real-world • Trial status • Chronic Lymphocytic Leukemia • Hematological Malignancies • Immunology • Leukemia • Oncology

Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma (clinicaltrials.gov) - P3 | N=53 | Completed | Sponsor: Children's Oncology Group | Active, not recruiting ➔ Completed | Trial completion date: Dec 2023 ➔ Dec 2022

Trial completion • Trial completion date • Ataxia • CNS Tumor • Movement Disorders • Neuroblastoma • Neuroendocrine Tumor • Oncology • Solid Tumor

IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS or FGFR-3 Antibodies (clinicaltrials.gov) - P2 | N=20 | Not yet recruiting | Sponsor: Loyola University | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Jun 2025 ➔ Jun 2026

Trial completion date • Trial primary completion date • Immunology • Pain

Study of Intravenous Immunoglobulin in Amnestic Mild Cognitive Impairment (clinicaltrials.gov) - P2 | N=52 | Completed | Sponsor: Sutter Health | Unknown status ➔ Completed

Trial completion • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • APOE • CSF P-tau

NGAM-13: Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (clinicaltrials.gov) - P3 | N=92 | Recruiting | Sponsor: Octapharma | Suspended ➔ Recruiting | Trial completion date: Jan 2023 ➔ Oct 2024 | Trial primary completion date: Jan 2023 ➔ Oct 2024

Enrollment open • Head-to-Head • Trial completion date • Trial primary completion date • Pediatrics • Psychiatry

NGAM-13: Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome (clinicaltrials.gov) - P3 | N=92 | Suspended | Sponsor: Octapharma | Recruiting ➔ Suspended

Head-to-Head • Trial suspension • Pediatrics • Psychiatry

PROSID: Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study) (clinicaltrials.gov) - P3 | N=240 | Recruiting | Sponsor: Octapharma | Trial completion date: Dec 2026 ➔ Oct 2024 | Trial primary completion date: Dec 2026 ➔ Oct 2024

Trial completion date • Trial primary completion date • Chronic Lymphocytic Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Oncology

NGAM-11: Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients (clinicaltrials.gov) - P3 | N=30 | Not yet recruiting | Sponsor: Octapharma | Trial completion date: Jun 2025 ➔ Jun 2026 | Trial primary completion date: Jun 2025 ➔ Dec 2025

Trial completion date • Trial primary completion date • Pain • Pediatrics