Baxter Submits Application for FDA Approval of GAMMAGARD LIQUID to Treat Multifocal Motor Neuropathy, a Degenerative Neurological Disorder

DEERFIELD, Ill.--(BUSINESS WIRE)--Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a supplemental biologics license application (sBLA) to the U.S. Food and Drug Administration (FDA) for approval of GAMMAGARD LIQUID 10% [Immune Globulin Infusion (Human)] for the treatment of multifocal motor neuropathy (MMN). The product, marketed as KIOVIG outside the United States and Canada, was approved for the MMN indication in Europe in 2011.

“Effective treatment options for MMN are limited and, if approved, GAMMAGARD LIQUID will be the first immunoglobulin treatment approved for MMN patients,” said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business. “This filing supports Baxter’s commitment to improving patient care, particularly by studying the use of our products in areas where treatment options are limited, such as chronic neurological diseases.”

MMN is associated with a progressive, asymmetric limb weakness mostly affecting the upper limbs, which can lead to significant difficulty with simple manual tasks. If left untreated, involuntary twitching. Nearly 60 percent of people with MMN are between 20 and 65 years of age at onset of the disease, and men are more frequently affected than women. The prevalence of MMN is estimated at one or two in approximately 100,000 people. As such, Baxter has been granted Orphan Drug Designation for this indication in the United States, which includes treatments for diseases that affect fewer than 200,000 people.

The filing is based on a Phase III, randomized, double-blind, placebo-controlled, cross-over, multi-center study of the efficacy, safety, and tolerability of GAMMAGARD LIQUID 10% in a total of 44 MMN patients. The study evaluated whether GAMMAGARD LIQUID was superior to placebo administration in the primary and secondary endpoints, including grip strength, and results supported filing for approval in the United States.

About GAMMAGARD LIQUID

GAMMAGARD LIQUID is indicated as replacement therapy for primary humoral immunodeficiency 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 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.

Prior to administering GAMMAGARD LIQUID, 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, GAMMAGARD LIQUID must be administered 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.

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.

GAMMAGARD LIQUID contains blood group antibodies which may act as hemolysins and induce in vivo coating of red blood cells (RBC) with immune globulin. Acute intravascular hemolysis has been reported, and delayed hemolytic anemia can develop due to enhanced RBC sequestration.

Non-cardiogenic pulmonary edema (TRALI) has been reported in patients following treatment with IGIV products, including GAMMAGARD LIQUID.

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.

Intravenous: The most serious adverse reaction seen during intravenous treatment in the clinical trials was two episodes of aseptic meningitis in one subject. The most common adverse reactions (observed in ≥5% of subjects) were headache, pyrexia, fatigue, rigors, nausea, chills, dizziness, vomiting, migraine headache, pain in extremity, urticaria, cough, pruritus, rash, and tachycardia.

Please review the GAMMAGARD LIQUID Prescribing Information for full prescribing details.

http://www.baxter.com/products/biopharmaceuticals/downloads/gamLIQUID_PI.pdf

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.

This release includes forward-looking statements concerning the potential use of GAMMAGARD LIQUID for the treatment of multifocal motor neuropathy. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: actions of regulatory bodies and other governmental authorities, including the FDA; satisfaction of regulatory and other requirements; and other risks identified in the company's most recent filing on Form 10-K and other SEC filings, all of which are available on the company's website. The company does not undertake to update its forward-looking statements.