PI Pivotal Trial Results—Efficacy

Young man

The efficacy and safety of Gammaplex 5% in PI was assessed in a 12-month study

  • Open-label, non-comparative study conducted at seven US centers evaluating Gammaplex 5% (immune globulin intravenous [human], 5% liquid) in the treatment of 50 adult patients with primary immunodeficiency (PID) and significant hypogammaglobulinemia1
  • Prior to starting Gammaplex 5%, patients were stable on a different IVIG for at least 3 months1
    • 43/50 patients (86%) were taking a 10% IVIG product2
  • Primary endpoint was the incidence of serious acute bacterial infections (SABIs). SABIs were defined as1:
    • Bacterial pneumonia
    • Bacteraemia or sepsis
    • Osteomyelitis/septic arthritis
    • Visceral abscess
    • Bacterial meningitis

Pharmacokinetic results1:

  • Mean IgG half-life was 41.1 days in a predefined subset of 24 patients
  • IgG trough levels were similar on Gammaplex 5% and on previous therapy
  • Median IgG trough concentrations after 15 weeks on Gammaplex 5% were within the range for subjects without PID, which previously has been shown to confer effective protection against infection in patients with PID

Gammaplex 5% is made from human plasma and may contain infectious agents, e.g., viruses and, theoretically, the Creutzfeldt-Jakob disease agent.

Over 12 months of treatment with Gammaplex 5%, no SABIs were reported1

  • The primary endpoint surpassed the FDA minimum requirement of <1.0 SABI per patient per year
  • 6/50 patients (12%) reported a SABI in the previous 6 months before switching to Gammaplex 5%
  • The most common adverse reactions reported in >5% of PI clinical trial subjects were headache, pyrexia, fatigue, nausea, hypertension, chills, myalgia, pain, and vomiting
Missed school or work

*This patient underwent lumbar disc surgery, experienced recurrent wheezing and had a coincidental squamous cell carcinoma (240 days off school/work).

References: 1. Moy JN, Scharenberg AM, Stein AR, et al. Clin Exp Immunol. 2010;162:510-515. 2. Data on File, BPL-GMX101-1008.

Important Safety Information for Gammaplex 5%

Gammaplex 5% (immune globulin intravenous [human], 5% liquid) is indicated for replacement therapy in primary humoral immunodeficiency (PI) in adults and pediatric patients two years of age and older. This includes, but is not limited to, the humoral immune defect in common variable immunodeficiency, X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

Gammaplex 5% is also indicated for the treatment of chronic immune thrombocytopenic purpura (ITP).

Thrombosis may occur with immune globulin products, including Gammaplex 5%. 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 immune globulin intravenous (IGIV) products, including Gammaplex 5%.

Patients predisposed to renal dysfunction include those with any 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 IGIV products containing sucrose. Gammaplex 5% does not contain sucrose.

For patients at risk of thrombosis, renal dysfunction or acute renal failure, administer Gammaplex 5% 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.

Gammaplex 5% is contraindicated in patients who have had a history of anaphylactic or severe systemic reactions to human immune globulin; a hereditary intolerance to fructose and in infants and neonates for whom sucrose or fructose tolerance has not been established; and IgA deficient patients with antibodies to IgA and a history of hypersensitivity.

In patients at risk of developing acute renal failure, monitor renal function, including blood urea nitrogen (BUN), serum creatinine and urine output. Hyperproteinemia, increased serum viscosity, and hyponatremia may occur in patients receiving IGIV therapy.

Aseptic meningitis syndrome (AMS) may occur infrequently with IGIV treatment. AMS usually begins within several hours to 2 days following IGIV treatment. Discontinuation of IGIV treatment has resulted in remission of AMS within several days without sequelae. AMS may occur more frequently in association with high doses (2 g/kg) and/or rapid infusion of IGIV.

Hemolysis and hemolytic anemia can develop subsequent to IGIV treatments. Patient risk factors that may be associated with development of hemolysis include high dose (>2 g/kg), non-O blood group, and underlying inflammatory state. Noncardiogenic pulmonary edema may occur in patients following IGIV treatment (i.e. transfusion-related acute lung injury [TRALI]). Monitor patients for pulmonary adverse reactions. If TRALI is suspected, test product and patient’s serum for anti-neutrophil antibodies.

Gammaplex 5% is made from human plasma and may contain infectious agents, e.g. viruses and, theoretically, the Creutzfeldt-Jakob disease agent. No cases of transmission of viral diseases or CJD have been associated with the use of Gammaplex 5%.

In clinical studies, the most common adverse reactions with Gammaplex 5% were headache, pyrexia, vomiting, fatigue, nausea, sinusitis, and nasal congestion. Serious adverse reactions observed in clinical trial subjects with primary immunodeficiencies were thrombosis and chest pain. Serious adverse reactions observed in clinical trial subjects with Immune Thrombocytopenic Purpura were headache, vomiting and dehydration.

Please see Full Prescribing Information for complete prescribing details.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch, or call 1-800-FDA-1088.

You may also call BPL at 1-844-4BPLUSA or email medinfo@bpl-us.com.