Primary Endpoint
Primary Study Design1
A 1-year, prospective, open-label, non-controlled, non-randomized, multicenter, Phase 3 study evaluated the efficacy, safety, and tolerability of PANZYGA in 51 adult and pediatric patients with PI at a maximum infusion rate of 8 mg/kg/min.
Key Inclusion Criteria
- Adult or pediatric patients with confirmed diagnosis of CVID or XLA
- Previous treatment with a commercial IVIg at a dose of 200-800 mg/kg every 21-28 days for at least six infusion intervals
- Evidence of an IgG trough level of ≥550 mg/dL at the previous two infusions before enrollment
- For female patients, a negative pregnancy test and use of a reliable contraceptive method for the study duration
Study Endpoints
Primary Endpoint
Rate of serious bacterial infections (SBIs) with treatment per patient per year
Supporting Efficacy Endpoints
Effects of treatment on quality-of-life (QoL) measures, including the number of days of work or school missed
Safety Assessments
- Occurrence of an infection of any kind of seriousness
- Time to resolution of infections
- Use of antibiotics
- Number of days of hospitalization
- Number of episodes of fever
Extension Study Design1,2
The extension study included 21 patients who completed the primary study and were previously treated with at least 3 infusions of PANZYGA at an infusion rate of 8 mg/kg/min without premedication.
During the extension study, the safety and tolerability of higher infusion rates (8 mg/kg/min up to 14 mg/kg/min) of PANYZGA for 3 months were assessed.
41%
(21/51)
of patients from the primary study were eligible for the extension study1
Primary Endpoint
PANZYGA successfully prevented serious bacterial infections (SBIs).
0.08 SBIs
per patient per year
Supporting Efficacy Endpoints1
Rates of other infections and absences from work of school were low.
3.7
infections per patient per year
3.6
absent days per patient per year
Safety Assessments1,2
There were limited hospitalizations or antibiotic use due to infection.
1
adult patient was hospitalized for 4 days
2.5
treatment episodes with antibiotics per adult patient per year
0.1
overall rate of days in hospital per patient per year
4.3
treatment episodes with antibiotics per adolescent patient per year
Safety & Tolerability
in PI Patients
Learn More
CVID=common variable immunodeficiency disorder;
PI=primary immunodeficiency;
XLA=X-linked agammaglobulinemia.
References: 1. Panzyga. Prescribing information. Octapharma USA, Inc.; 2021. 2. Borte M, Melamed IR, Pulka G, et al. Efficacy and safety of humanintravenous immunoglobulin 10%(Panzyga®) in patients with primary immunodeficiency diseases: a two-stage, multicenter, prospective, open-labelstudy. JClin Immunol. 2017; 37(6):603-612. doi: 10.1007/s10875-017-04244r-4
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR PANZYGA® IMMUNE GLOBULIN INTRAVENOUS (HUMAN) – IFAS 10% LIQUID PREPARATION
BOXED WARNING: THROMBOSIS, RENAL DYSFUNCTION, AND ACUTE RENAL FAILURE
Please click here for Full Prescribing Information, including BOXED WARNING.
- Thrombosis may occur with immune globulin intravenous (IVIg) products, including Panzyga. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling central vascular catheters, hyperviscosity, and cardiovascular risk factors. Thrombosis may occur in the absence of known risk factors.
- Renal dysfunction, acute renal failure, osmotic nephrosis, and death may occur in predisposed patients who receive IVIg products, including Panzyga. Patients predisposed to renal dysfunction include those with a degree of pre-existing renal insufficiency, diabetes mellitus, age greater than 65, volume depletion, sepsis, paraproteinemia, or patients receiving known nephrotoxic drugs. Renal dysfunction and acute renal failure occur more commonly in patients receiving IVIg products containing sucrose. Panzyga does not contain sucrose.
- For patients at risk of thrombosis, renal dysfunction, or acute renal failure, administer Panzyga at the minimum dose and 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.
See Full Prescribing Information, Warnings and Precautions (5.2, 5.4)
Indications and Usage
Panzyga (Immune Globulin Intravenous [Human] – ifas) is indicated for the treatment of primary humoral immunodeficiency (PI) in patients 2 years of age and older; this includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency, X-linked agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies; chronic immune thrombocytopenia (cITP) in adults to raise platelet counts to control or prevent bleeding; and chronic inflammatory demyelinating polyneuropathy (CIDP) in adults to improve neuromuscular disability and impairment.
Contraindications
Panzyga is contraindicated in patients who have a history of severe systemic hypersensitivity reactions, such as anaphylaxis, to human immunoglobulin and in IgA-deficient patients with antibodies against IgA and history of hypersensitivity.
Warnings and Precautions
- Monitor renal function, including blood urea nitrogen and serum creatinine, and urine output in patients at risk of developing acute renal failure.
- Hyperproteinemia, increased serum osmolarity, and hyponatremia may occur in patients receiving Panzyga.
- Aseptic meningitis syndrome may occur in patients receiving Panzyga, especially with high doses or rapid infusion.
- Hemolysis that is either intravascular or due to enhanced red blood cell sequestration can develop subsequent to Panzyga treatments. Risk factors for hemolysis include high doses and non-O-blood group. Closely monitor patients for hemolysis and hemolytic anemia.
- Monitor patients for pulmonary adverse reactions (transfusion-related acute lung injury [TRALI]).
- Monitor blood pressure prior to, during, and following Panzyga infusion.
- Carefully consider the relative risks and benefits before prescribing the high dose regimen (for cITP) in patients at increased risk of volume overload.
- Panzyga is made from human plasma and may contain infectious agents, e.g. viruses and theoretically, the Creutzfeldt-Jakob disease agent.
Adverse Reactions
- PI – The most common adverse reactions reported in greater than 5% of subjects were: headache, nausea, fever, fatigue, and abdominal pain.
- cITP in adults – The most common adverse reactions reported in greater than 5% of subjects were: headache, fever, nausea, vomiting, dizziness, and anemia.
- CIDP in adults – The most common adverse reactions reported in greater than 5% of subjects were: headache, fever, dermatitis, and blood pressure increase.
The risk information provided here is not comprehensive; See full Prescribing Information and Boxed Warning for Panzyga.
To report suspected adverse reactions, contact Octapharma USA, Inc. at 1-866-766-4860 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR PANZYGA® IMMUNE GLOBULIN INTRAVENOUS (HUMAN) – IFAS 10% LIQUID PREPARATION
BOXED WARNING: THROMBOSIS, RENAL DYSFUNCTION, AND ACUTE RENAL FAILURE
Please click here for Full Prescribing Information, including BOXED WARNING.
- Thrombosis may occur with immune globulin intravenous (IVIg) products, including Panzyga. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling central vascular catheters, hyperviscosity, and cardiovascular risk factors. Thrombosis may occur in the absence of known risk factors.
- Renal dysfunction, acute renal failure, osmotic nephrosis, and death may occur in predisposed patients who receive IVIg products, including Panzyga. Patients predisposed to renal dysfunction include those with a degree of pre-existing renal insufficiency, diabetes mellitus, age greater than 65, volume depletion, sepsis, paraproteinemia, or patients receiving known nephrotoxic drugs. Renal dysfunction and acute renal failure occur more commonly in patients receiving IVIg products containing sucrose. Panzyga does not contain sucrose.
- For patients at risk of thrombosis, renal dysfunction, or acute renal failure, administer Panzyga at the minimum dose and 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.
See Full Prescribing Information, Warnings and Precautions (5.2, 5.4)
Indications and Usage
Panzyga (Immune Globulin Intravenous [Human] – ifas) is indicated for the treatment of primary humoral immunodeficiency (PI) in patients 2 years of age and older; this includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency, X-linked agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies; chronic immune thrombocytopenia (cITP) in adults to raise platelet counts to control or prevent bleeding; and chronic inflammatory demyelinating polyneuropathy (CIDP) in adults to improve neuromuscular disability and impairment.
Contraindications
Panzyga is contraindicated in patients who have a history of severe systemic hypersensitivity reactions, such as anaphylaxis, to human immunoglobulin and in IgA-deficient patients with antibodies against IgA and history of hypersensitivity.
Warnings and Precautions
- Monitor renal function, including blood urea nitrogen and serum creatinine, and urine output in patients at risk of developing acute renal failure.
- Hyperproteinemia, increased serum osmolarity, and hyponatremia may occur in patients receiving Panzyga.
- Aseptic meningitis syndrome may occur in patients receiving Panzyga, especially with high doses or rapid infusion.
- Hemolysis that is either intravascular or due to enhanced red blood cell sequestration can develop subsequent to Panzyga treatments. Risk factors for hemolysis include high doses and non-O-blood group. Closely monitor patients for hemolysis and hemolytic anemia.
- Monitor patients for pulmonary adverse reactions (transfusion-related acute lung injury [TRALI]).
- Monitor blood pressure prior to, during, and following Panzyga infusion.
- Carefully consider the relative risks and benefits before prescribing the high dose regimen (for cITP) in patients at increased risk of volume overload.
- Panzyga is made from human plasma and may contain infectious agents, e.g. viruses and theoretically, the Creutzfeldt-Jakob disease agent.
Adverse Reactions
- PI – The most common adverse reactions reported in greater than 5% of subjects were: headache, nausea, fever, fatigue, and abdominal pain.
- cITP in adults – The most common adverse reactions reported in greater than 5% of subjects were: headache, fever, nausea, vomiting, dizziness, and anemia.
- CIDP in adults – The most common adverse reactions reported in greater than 5% of subjects were: headache, fever, dermatitis, and blood pressure increase.
The risk information provided here is not comprehensive; See full Prescribing Information and Boxed Warning for Panzyga.
To report suspected adverse reactions, contact Octapharma USA, Inc. at 1-866-766-4860 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.