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 for the treatment of 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 rapidly raise platelet counts to prevent bleeding in patients with chronic immune thrombocytopenic purpura (ITP).
WARNING: Renal dysfunction, acute renal failure, osmotic nephrosis, and death may be associated with the administration of Immune Globulin Intravenous (Human) (IVIg) products in predisposed patients. Administer IVIg products at the minimum infusion rate possible. Renal dysfunction and acute renal failure occur more commonly in patients receiving IVIg products containing sucrose. Privigen does not contain sucrose. See full prescribing information for complete boxed warning.
Privigen is contraindicated in patients who have had an anaphylactic or severe systemic reaction to the administration of human immune globulin, in patients with hyperprolinemia, and in IgA-deficient patients with antibodies to IgA and a history of hypersensitivity.
In patients at risk for developing renal failure, monitor urine output and renal function, including blood urea nitrogen and serum creatinine. Thrombotic events have been reported with Privigen and other IVIg treatments. Monitor patients with risk factors for thrombotic events, including a history of atherosclerosis, multiple cardiovascular risk factors, advanced age, impaired cardiac output, hypercoagulable disorders, prolonged periods of immobilization, and/or known or suspected hyperviscosity.
Aseptic meningitis syndrome (AMS) may occur infrequently with Privigen and other IVIg treatments; AMS may occur more frequently with high doses and/or rapid infusion of IVIg. Hemolysis, hemolytic anemia, and pulmonary adverse events have also been reported. There have been reports of noncardiogenic pulmonary edema in patients administered IVIg. If transfusion-related acute lung injury is suspected, test product and patient for antineutrophil antibodies.
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, the most common adverse reactions with Privigen were headache, pain, nausea, pyrexia/hyperthermia, fatigue, and chills. Anemia was an additional common adverse reaction in patients being treated with Privigen for chronic ITP. The most serious adverse reactions in chronic ITP patients were aseptic meningitis syndrome in one subject and hemolysis in eight subjects.
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.