DEERFIELD, Ill., June 25, 2012 - Baxter International Inc. (NYSE:BAX) today announced that the U.S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID 10% [Immune Globulin Infusion (Human)] as a treatment for multifocal motor neuropathy (MMN). This is the first immunoglobulin treatment approved for MMN patients in the United States, and it was approved for use with MMN patients in Europe in 2011.
MMN is associated with a progressive, asymmetric limb weakness mostly affecting the upper limbs, which can lead to significant difficulty with simple manual tasks. Nearly 80 percent of people with MMN are between 20 and 50 years of age at onset of the disease, and men are more frequently affected than women. Baxter has been granted Orphan Drug Designation for this indication in the United States, as the prevalence of MMN is estimated at between one or two people for approximately 100,000 individuals.
"Multifocal motor neuropathy, known as MMN, is a challenging neuromuscular disease to diagnose and manage, and patients have had limited treatment options. With the introduction of GAMMAGARD LIQUID as an FDA approved treatment option, we're able to offer patients a proven therapy to help them improve their function in their hands and feet," said Said R. Beydoun, M.D., FAAN, professor of neurology at the Keck Medical Center of USC, University of Southern California and investigator on the MMN clinical trial.
"The regulatory approval of GAMMAGARD LIQUID for the treatment of MMN is an important milestone for Baxter as we continue to build our presence in the immunoglobulin market and expand into rare diseases in neurology. With GAMMAGARD, we can offer patients with MMN a therapeutic option that has been well studied and used with primary immunodeficiency patients for many years," said Ludwig Hantson, Ph.D., president of Baxter's BioScience business.
The approval was based on the results of a randomized, double-blind, placebo controlled, cross-over study conducted to evaluate the efficacy and safety/tolerability of GAMMAGARD LIQUID in 44 adult subjects with MMN. The pivotal clinical study results were presented during the American Academy of Neurology annual meeting in April 2012. The two co-primary endpoints were grip strength in the more affected hand and disability, as measured by Guy's Neurological Disability Scale (which measures the patient's ability to perform daily tasks such as zippering, buttoning, tying shoe laces, washing and feeding).
During GAMMAGARD LIQUID treatment, the difference in relative change in mean grip strength in the more affected hand was 22.94% compared to placebo. A greater proportion of patients who received placebo experienced deterioration compared to those receiving GAMMAGARD LIQUID (35.7% vs.11.9%, respectively). The differences in the outcomes of the co-primary endpoints were statistically significant. In addition, the majority of patients (69%) required an accelerated switch to GAMMAGARD LIQUID due to functional deterioration during the placebo period.
The most common adverse reactions (observed in ≥5% of subjects) during infusion with GAMMAGARD LIQUID were headache, chest discomfort, muscle spasms, muscular weakness, nausea, oropharyngeal pain, and pain in extremity. The serious adverse reactions were pulmonary embolism and blurred vision which were judged to be treatment-related. No deaths or unexpected serious adverse events related to study product occurred.
About GAMMAGARD LIQUID
GAMMAGARD LIQUID is indicated as replacement therapy for primary humoral immunodeficiency (PI) in adult and pediatric patients two years of age or older. This includes, but is not limited to, common variable immunodeficiency (CVID), X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.
GAMMAGARD LIQUID is indicated as a maintenance therapy to improve muscle strength and disability in adult patients with Multifocal Motor Neuropathy (MMN).
GAMMAGARD LIQUID was originally approved by the U.S. Food and Drug Administration (FDA) in September 2005. Also known as KIOVIG outside the United States and Canada, it is approved in 51 countries worldwide.
IMPORTANT RISK INFORMATION
- Renal dysfunction, acute renal failure, osmotic nephrosis, and death may occur with immune globulin intravenous (IGIV) products in predisposed patients. 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. GAMMAGARD LIQUID does not contain sucrose.
- For patients at risk of renal dysfunction or failure, administer GAMMAGARD LIQUID at the minimum infusion rate practicable.
Ensure that patients with pre-existing renal insufficiency are not volume depleted. For patients over 65 years of age or judged to be at risk for renal dysfunction or thrombotic events, administer GAMMAGARD LIQUID at the minimum infusion rate practicable. In such cases, the maximal rate should be less than 3.3 mg/kg/min (< 2mL/kg/hr), and consider discontinuation of administration if renal function deteriorates.
GAMMAGARD LIQUID is contraindicated in patients who have had a history of anaphylactic or severe systemic hypersensitivity reactions to the administration of human immune globulin.
GAMMAGARD LIQUID is contraindicated in IgA-deficient patients with antibodies to IgA and a history of hypersensitivity. Anaphylaxis has been reported with the intravenous use of GAMMAGARD LIQUID and is theoretically possible following subcutaneous administration.
Severe hypersensitivity reactions may occur, even in patients who had tolerated previous treatment with human normal immune globulin. In case of hypersensitivity, discontinue GAMMAGARD LIQUID infusion immediately and institute appropriate treatment.
Hyperproteinemia, increased serum viscosity, and hyponatremia may occur in patients receiving GAMMAGARD LIQUID.
Thrombotic events, including myocardial infarction, cerebral vascular accident, deep vein thrombosis, and pulmonary embolism have been reported in association with intravenous use of GAMMAGARD LIQUID. Thrombotic events have also been reported with subcutaneous administration of immune globulin. Patients at risk for thrombotic events include those with a history of atherosclerosis, multiple cardiovascular risk factors, advanced age, impaired cardiac output, coagulation disorders, prolonged periods of immobilization, obesity, diabetes mellitus, acquired or inherited thrombophilic disorder, a history of vascular disease, or a history of a previous thrombotic or thromboembolic event.
Aseptic Meningitis Syndrome may occur with IGIV treatment, and has been reported with intravenous use of GAMMAGARD LIQUID. Discontinuation of IGIV treatment has resulted in remission of AMS within several days without sequelae. The syndrome usually begins within several hours to two days following IGIV treatment.
GAMMAGARD LIQUID contains blood group antibodies that may act as hemolysins and induce in vivo coating of red blood cells (RBC) with immune globulin. Delayed hemolytic anemia can develop subsequent to GAMMAGARD LIQUID therapy due to enhanced RBC sequestration; acute hemolysis, consistent with intravascular hemolysis, has been reported. The following risk factors may be related to the development of hemolysis: high doses (e.g., ≥2 grams/kg, single administration or divided over several days) and non-O blood group. Underlying inflammatory state in an individual patient may increase the risk of hemolysis but its role is uncertain. Monitor patients for clinical signs and symptoms of hemolysis, particularly patients with risk factors noted above.
Non-cardiogenic pulmonary edema (TRALI) has been reported in patients following treatment with IGIV products, including GAMMAGARD LIQUID. Monitor patients for pulmonary adverse reactions.
GAMMAGARD LIQUID is made from human plasma. It may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and theoretically, the classic Creutzfeldt-Jakob disease agent. This also applies to unknown or emerging viruses and other pathogens. No cases of transmission of viral diseases or vCJD have been associated with GAMMAGARD LIQUID.
PI: Intravenous: The serious adverse reaction seen during intravenous treatment in the clinical trials for PI aseptic meningitis. The most common adverse reactions for PI (observed in ≥5% of subjects) were headache, fatigue, pyrexia, nausea, chills, rigors, pain in extremity, diarrhea, migraine, dizziness, vomiting, cough, urticaria, asthma, pharyngolarynegeal pain, rash, arhralgia, myalgia, oedema peripheral, pruritus, and cardiac murmur.
PI: Subcutaneous: No serious adverse reactions were observed during the clinical trial for subcutaneous treatment. The most common adverse reactions during subcutaneous treatment (observed in ≥5% of PI subjects) were Infusion site (local) event, headache, fatigue, heart rate increased, pyrexia, abdominal pain upper, nausea, vomiting, asthma, blood pressure systolic increased, diarrhea, ear pain, aphthous stomatitis, migraine, oropharyneal pain, and pain in extremity.
MMN: The serious adverse reactions in the clinical trial for MMN were pulmonary embolism and blurred vision. The most common adverse reactions for MMN (observed in ≥5% of subjects) were headache, chest discomfort, muscle spasms, muscular weakness, nausea, oropharyneal pain, and pain in extremity.
Please review the GAMMAGARD LIQUID Prescribing Information for full prescribing details.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.