DEERFIELD, Ill., Apr 27, 2012 (BUSINESS WIRE) -- Baxter International Inc. /quotes/zigman/219387/quotes/nls/bax BAX +0.48% 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 (

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 greater-than or equal to 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.

SOURCE: Baxter International Inc.

        
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Copyright Business Wire 2012

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Frieda Brepoels MEP

CEAPIR vice president Mark Murphy

CEAPIR vice president Lars Engberg

Belgian MEP Frieda Brepoels has called for more to be done to enhance patient choice and access to treatment for people suffering from kidney disease.

Brepoels, speaking at an event in the European parliament on Thursday, described the disease as a "silent epidemic" and urged EU and national policymakers as well as healthcare professionals to "work together" to make sure that those suffering from end-stage kidney disease receive, "the right treatment at the right time".

She told the debate, jointly organised by the Parliament Magazine and the European kidney patients' federation (CEAPIR), that incidence of the disease, which can have a severely debilitating effect on sufferers, is set to double over the next decade.

Information and treatment choice

The roundtable debate heard evidence from several speakers involved in the production of a major Europe-wide survey highlighting widespread differences in the provision of information and treatment choice for kidney patients.

The CEAPIR survey, entitled, 'Unequal treatment for kidney patients in Europe', found that a quarter of end-stage kidney disease patients did not feel involved in the decisions made about their treatment choices.

It also found that there were stark differences and inequalities of treatment options across the 12 European countries that took part, with only 15 per cent of German patients surveyed saying they felt that they had received proper training or education on managing the disease against three quarters of those surveyed in Finland.

Brepoels, a member of the parliament's public health and food safety committee, said, "It goes without saying, that chronic kidney disease and its treatment have an enormous impact on patients' lives; it also entails significant socio-economic costs.

"Like many other chronic diseases, kidney disease is treatable, but not curable. Unfortunately, we see though that many patients are only referred to a kidney specialist when confronted with kidney failure and requiring dialysis. At this stage preventive measures are no longer effective and a lot of vulnerable time is lost."

Brepoels added, "Chronic kidney disease is a silent epidemic; in many cases affected persons are not aware of their condition because the disease often develops without symptoms."

Treatment options

Transplantation is seen as the 'gold standard' for treating the disease and is recognised as the best option in terms of quality of life and patient survival as well as the most cost-effective treatment for national healthcare authorities.

However because of a shortage of donor organs and suitable living donors, kidney dialysis is still the most commonly used treatment.

However, despite the availability of a home treatment procedure called peritoneal dialysis (PD) almost 90 per cent of patients currently use Haemodialysis (HD), usually at a hospital or clinic.

From a patient perspective, PD is seen as more flexible allowing patients to take a greater role in managing their care.

The need for a survey on patient choice

"Patient involvement has a positive effect on quality of life and patient satisfaction," CEAPIR vice president Lars Engberg told participants at the event.

"Patients have a different perspective, so their involvement can affect their clinical outcome and help complement the knowledge of health professionals."

"A patient survey, like the one being discussed today, is one way of involving patients and allows us to focus on areas of action within the heath service".

Engberg added that polls such as the recent CEAPIR survey can especially benefit patients as the decisions they make on their treatment could have a "very strong impact" on their daily lives.

Hard data on treatment options was essential if organisations like CEAPIR wanted to better promote the interests of kidney patients said Engberg. "We need to understand if we want to better represent patients."

The debate heard of the moving experience of Peter Fahy, a 20-year-old Dublin-based kidney patient who spoke of the way the disease had transformed his life.

Fahy opted for home-based PD dialysis (normally done as he sleeps) as he felt it would be less disruptive and allow him to lead a reasonably normal life as a teenager at home rather than involve several trips a week to a hospital.

He said, "I have been receiving treatment since the age of 15 but just hope I can lead as normal a life as possible."

"It's important that people are informed of all the options so they can choose a treatment that suits their lifestyle."

Findings and challenges

Kajsa Wilhelmsson, a senior councillor with PA firm Edelman, presented details of the survey, which was supported by Baxter Healthcare, saying that it illustrated the need for "improvement" in most countries.

The report found that access to renal care for kidney disease patients was "unequal" and "erratic" with one in five patients polled saying they had not received information they found useful on dialysis treatment options.

One in three patients surveyed said they were not able to choose their treatment method or were unsure about how much their choice counted, while less than half said they believed that they were only told about alternative treatment options while already on dialysis.

The key challenges identified by the poll were, said Wilhelmsson, "early detection, equal quality, access, information and the education of patients".

An EU wide treatment register

Further comment came from Mark Murphy, also a vice president of CEAPIR, who called for more effective monitoring of the treatment available to patients, including a possible mandatory, EU-wide register.

He said, "There are significant differences in the cost of treatment for kidney patients between member states and this is something we want to see an end of."

Murphy added that the cheapest treatment was transplantation, which he said cost around the same as a year's worth of dialysis. However, there are also sizeable differences in the cost of dialysis treatment.

Around 90 per cent of dialysis patients use HD, which is mainly performed in hospitals. Murphy said that studies have proven that between 35-55 per cent would prefer to be treated at home if given the choice.

"EU member states are spending around two per cent of their total healthcare budget on dialysis treatment, yet dialysis patients make up only around 0.2 per cent of the population. National governments may not be spending their budgets wisely."

"Home treatment is cheaper and PD is significantly cheaper because it's done at home by patients"

Murphy highlighted a number of what he called "barriers to optimal care and patient choice", including affordability, accountability, health care professional and education, commercial bias geographical complexities, organ donation culture and the structural management of health services.

On accountability, Murphy called for an EU directive on mandatory reporting to a European Renal registry which would help set Europe-wide standards of quality for renal replacement therapy (RRT).

He argued that a mandatory register already exists for organ transfers across the EU, but, "we need to know the EU 27 numbers on RRT. A register has to become mandatory, there is a real need for good data."

Education and support

Murphy also called on the EU to take the lead in educating healthcare professionals in identifying symptoms and supporting those countries reporting low prevalence of kidney disease.

He also warned against what he called commercial bias within the prescribing of treatments linked to services supplying those treatments for patients, saying, "we need to recognise and investigate commercial interests [in treatment choices for kidney patients]. We don't let doctors prescribe and sell medicines, but do let doctors prescribe dialysis treatment and profit from that treatment."

Irish MEP Marian Harkin also warned of the dangers of commercial bias, saying that there was a need to ensure that there was no conflict of interest surrounding the running of treatment clinics.

She said there was "great interest" in the issue of patient choice and access to treatment and in "what role [EU policymakers] can play" in improving patient advocacy.

She added that "there is a need for good, robust information and perhaps we can add value by looking into issues such as whether there is a possibility to access funds for more research at European level through the EU's seventh research framework programme".

Closing the debate, Murphy said, "We have come a long way on [patient choices and access to treatment], but there is still a long way to go.

"Today's discussions in the European parliament mark a big step towards equipping patients with information and securing patient choice and equal access to renal care, both at European and national levels."

Further reading here

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