Healthcare Industry News: orphan drug designation
News Release - April 27, 2012
Baxter Presents Efficacy and Safety Data Evaluating GAMMAGARD LIQUID for Multifocal Motor Neuropathy at AAN Annual MeetingDEERFIELD, Ill.--(Healthcare Sales & Marketing Network)--Baxter International Inc. (NYSE:BAX ) today announced the presentation of Phase III clinical data evaluating the safety and efficacy of GAMMAGARD LIQUID 10% [Immune Globulin Infusion (Human)] for the treatment of multifocal motor neuropathy (MMN). The data were presented during a Clinical Trials Forum at the 64th annual meeting of the American Academy of Neurology (AAN) in New Orleans, La., and formed the basis for a regulatory filing seeking an indication to treat the condition.
The Phase III, randomized, double-blind, placebo-controlled, cross-over, multi-center study evaluated whether GAMMAGARD LIQUID was superior to placebo in preserving muscle grip strength and preventing progression of disability in 44 patients with MMN. Patients completed five study phases, each 12 weeks long (three open-label phases with GAMMAGARD LIQUID and two double-blind, cross-over treatment phases with GAMMAGARD LIQUID or placebo), with an average monthly dose of 1.2 g/kg body weight. This is the largest randomized clinical trial of patients with MMN to date. MMN is associated with a progressive, asymmetric limb weakness most often affecting the upper limbs, which can lead to significant difficulty with simple manual tasks.
GAMMAGARD LIQUID met its two primary efficacy endpoints, demonstrating a 3.75 percent increase in mean grip strength of the more affected hand during treatment, as compared to a 31.38 percent decrease in mean grip strength with placebo administration. The study also found that a greater proportion of patients who received placebo experienced progressive disability, as assessed by the Guy’s Neurological Disability Score compared to those receiving GAMMAGARD LIQUID (35.7% vs. 11.9%, respectively).
If study participants experienced intolerable interference with daily activities, the study allowed them to switch from placebo to GAMMAGARD LIQUID. The majority of patients on placebo (69%) required an accelerated switch before 12 weeks, with some in as few as seven days.
"MMN is a debilitating progressive disease and this development program helped assess the potential for new uses for GAMMAGARD LIQUID. If approved by the regulatory authorities, GAMMAGARD LIQUID will become the first immunoglobulin treatment available for patients with MMN in the United States," said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business.
No deaths or unexpected serious adverse events related to study product occurred. The most commonly reported adverse reactions (reported in five or fewer percent of subjects) were headache and muscular weakness. The proportion of infusions associated with headache was 2.1 percent with GAMMAGARD LIQUID and 2.3 percent with placebo. The proportion of infusions associated with muscular weakness was 0.4 percent with GAMMAGARD LIQUID and 0.8 percent with placebo.
The prevalence of MMN is estimated at one in approximately 100,000 people. Baxter has been granted orphan drug designation in pursuit of this indication in the United States, which includes treatments for diseases that affect fewer than 200,000 people. Baxter filed a supplemental biologics license application with the U.S. Food and Drug Administration (FDA) in December 2011 seeking an indication for GAMMAGARD LIQUID to include MMN. The product, marketed as KIOVIG outside the United States and Canada, was approved for the MMN indication in Europe in 2011.
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 and 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.
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.
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, 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: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; and other risks identified in Baxter’s most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter’s website. Baxter does not undertake to update its forward-looking statements.
Issuer of this News Release is solely responsible for its
Please address inquiries directly to the issuing company.