Gammaplex® Approved For Use In Pediatric Patients
Gammaplex [Immune Globulin Intravenous (Human) 5% Liquid] Approved in Patients
2 years and Older with Primary Immunodeficiencies
ELSTREE, UK AND DURHAM, NC – August 6, 2015 - Bio Products Laboratory Limited (BPL), a leading manufacturer of plasma-derived protein therapies, today announced the approval of Gammaplex® (Immune Globulin Intravenous [human], 5% liquid) for pediatric patients two years of age and older who have primary humoral immunodeficiencies. The approval was based on data submitted to the U.S. Food and Drug Administration (FDA) as part of a post-marketing commitment following approval of Gammaplex for replacement therapy in adults in 2009.
Primary immunodeficiencies are a constellation of immune disorders that can negatively impact the body’s ability to fight infection. In some primary immunodeficiencies, the immune system does not manufacture adequate quantities of antibodies such as immune globulin G. This indication 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.
According to Eric Wolford, PharmD, Vice President of Global Medical for BPL, “The approval of Gammaplex for pediatric use in primary immunodeficiencies represents another milestone in BPL’s continuing efforts to provide appropriate therapies to patients with rare diseases.”
Data supporting the approval was generated from a clinical study involving 25 children and adolescents with primary immunodeficiencies aged 3-16 years who were treated with Gammaplex for 12 months. The study’s primary efficacy endpoint was the incidence of serious, acute bacterial infections (SABIs) as defined by the FDA. Secondary objectives assessed safety and tolerability. Throughout the course of the study, two SABIs of pneumonia were reported, resulting in an annual SABI event rate of 0.09 (upper, 1-sided 99% CI, 0.36), well below the maximum SABI event rate of 0.5 per subject required for approval.
Fourteen subjects (56%) had an adverse reaction at some time during the clinical trial that was considered product-related. Of these 14 subjects, two had adverse reactions that were considered definitely related to Gammaplex including headache, fatigue, and myalgia. The most common adverse reactions, occurring in ≥5% of subjects were dyspnea (2/25 subjects, 8%), otitis media acute (2/25 subjects, 8%), and tonsillar disorder (2/25 subjects, 8%). Two subjects reported a serious adverse event of lobar pneumonia. Neither serious adverse reaction was considered related to Gammaplex, and neither met FDA-defined SABI criteria. No subjects withdrew from the study due to adverse events.
Gammaplex 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 also in IgA deficient patients with antibodies to IgA and a history of hypersensitivity.
Please see the additional Important Safety Information about Gammaplex set forth below, and the full prescribing information at
About Gammaplex Gammaplex is indicated for replacement therapy in adults and children 2 years of age and older with primary humoral immunodeficiency (PI). 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 is also indicated for the treatment of chronic immune thrombocytopenic purpura (ITP).
Important Safety Information
Thrombosis may occur with immune globulin products, including Gammaplex. 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.
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 does not contain sucrose.
For patients at risk of thrombosis, renal dysfunction or acute renal failure, administer Gammaplex 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 is contraindicated in patients who have had a history of anaphylactic or severe systemic reactions to human immune globulin; an 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.
Thrombotic events may occur following treatment with immune globulin products, including Gammaplex. Monitor patients with known risk factors for thrombotic events; consider baseline assessment of blood viscosity for those at risk of hyperviscosity.
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 (TRALI). If TRALI is suspected, test product and patient’s serum for anti-neutrophil antibodies.
Gammaplex 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.
In clinical studies, the most common adverse reactions with Gammaplex were headache, pyrexia, vomiting, fatigue, pain, nausea, hypertension, chills and myalgia. 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.
About Bio Products Laboratory Limited
Bio Products laboratory Limited (BPL) is a leading manufacturer of plasma-derived protein therapies with global headquarters in Elstree, England, US headquarters in Durham, NC, and a presence in more than 45 countries worldwide. The company has over 60 years of experience developing and manufacturing plasma-derived therapies since being established as part of the Lister Institute in 1950, and currently markets Gammaplex in the US and a wide range of products outside the US, including coagulation factors, human immunoglobulins, and albumin. BPL is committed to continued investment in research and development to maintain its key position as a reliable supplier of high-quality products to patients and healthcare providers worldwide.