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.
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, 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.
