Efficacy and Safety in Patients with Primary Immunodeficiency Disease (PIDD)
The efficacy, safety, and pharmacokinetics of Privigen in primary immunodeficiency
disease were investigated in a prospective, open-label, single-arm, multicenter,
phase III study. The study was performed in 80 subjects with X-linked agammaglobulinemia
(XLA) (21 males) or common variable immune deficiency (CVID) (34 females and 25
males) who had been receiving intravenous immunoglobulin (IVIg) replacement therapy
for at least six months prior to the start of the study.6
Patients received Privigen at individually adjusted doses ranging from 200 mg/kg
to 888 mg/kg for 12 months.6
Efficacy
As shown below, the annual rate of acute serious bacterial infections (aSBIs)
per subject (defined as pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis,
bacterial meningitis, and visceral abscess) was 0.08 with an upper one-sided 99%
confidence interval of 0.182 and thus clearly below the predefined threshold of 1.0 mandated
by the FDA. When all infections were considered, the annual rate of infection per
subject was 3.55, which is consistent with published reports of other IVIg products.6
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Annual Rate of Infections (per Subject Year) During Clinical Trials*†
* Results from case report forms.
† Upper one-sided 99% confidence interval: 0.203.
‡ aSBIs defined as pneumonia, bacterial meningitis, bacteremia/septicemia,
osteomyelitis/septic arthritis, and visceral abscess.
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Safety
In clinical studies for PIDD, the most common adverse reactions with Privigen were headache,
pain, nausea, fatigue, and chills.6
- 97% of related adverse events were non-serious
- 95% of 1,038 infusions were given without premedication
Please see the Important Safety Information and
full Prescribing Information.
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Important Safety Information
Immune Globulin Intravenous (Human), 10% Liquid, Privigen® is indicated as replacement therapy for patients with primary immunodeficiency (PI) associated with defects in humoral immunity, including but not limited to common variable immunodeficiency (CVID), X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. Privigen is also indicated to raise platelet counts in patients with chronic immune thrombocytopenic purpura (ITP).
WARNING: Use of Immune Globulin Intravenous (IVIg) products, particularly those containing sucrose, have been associated with renal dysfunction, acute renal failure, osmotic nephropathy, and death. Privigen does not contain sucrose. Administer Privigen at minimum rate practicable in patients at risk of renal dysfunction or acute renal failure. At-risk patients include those with preexisting renal insufficiency, diabetes mellitus, volume depletion, sepsis, or paraproteinemia; over 65 years of age; or receiving known nephrotoxic drugs. See full prescribing information for complete boxed warning.
Privigen is contraindicated in patients with history of anaphylactic or severe systemic reaction to human immune globulin, in patients with hyperprolinemia, and in IgA-deficient patients with antibodies to IgA and history of hypersensitivity.
Monitor patient vital signs throughout infusion of Privigen. In cases of severe hypersensitivity or anaphylactic reactions, discontinue administration and institute appropriate medical treatment. In patients at risk for developing renal failure, monitor urine output and renal function, including blood urea nitrogen and serum creatinine. Also monitor patients with risk factors for thrombotic events; consider baseline assessment of blood viscosity for those at risk of hyperviscosity.
Patients could experience increased serum viscosity, hyperproteinemia or hyponatremia; infrequently, aseptic meningitis syndrome (AMS) may occur (most often with high doses and/or rapid IVIg infusion). There have been reports of IVIg-related hemolysis, hemolytic anemia, and pulmonary adverse events, including transfusion-related acute lung injury (TRALI). Avoid high-dose regimen where fluid volume is of concern.
Privigen is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.
In clinical studies of patients being treated with Privigen for PI, the most serious adverse reaction was hypersensitivity (one subject). Adverse reactions observed in >5% of subjects with PI were headache, pain, nausea, fatigue, chills, vomiting, joint swelling/effusion, pyrexia, and urticaria.
In clinical studies of patients being treated with Privigen for chronic ITP, the most serious adverse reactions were AMS (one subject) and hemolysis (eight subjects). Adverse reactions seen in >5% of subjects with chronic ITP were headache, pyrexia/hyperthermia, positive DAT, anemia, vomiting, nausea, increases in conjugated and unconjugated bilirubin, hyperbilirubinemia, and increased blood lactate dehydrogenase.
Treatment with Privigen might interfere with a patient's response to live virus vaccines and could lead to misinterpretation of serologic testing.
For more information about Privigen, please see full prescribing information.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.