LONDON, ENGLAND, June 24, 2011 - Baxter International Inc. (NYSE:BAX) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for extension of the therapeutic indications of KIOVIG to include a new indication for multifocal motor neuropathy (MMN), a severe, debilitating disorder requiring lifelong treatment. With adoption by the European Commission, Baxter will receive marketing authorization for KIOVIG MMN indication in all European Union (EU) Member States- the first centrally-licensed indication for an immunoglobulin preparation for MMN.
MMN is a rare autoimmune disorder characterized by progressive weakness in the limbs, leading to significant difficulty with simple manual tasks.1 If left untreated, MMN often progresses to more severe weakness, including muscle atrophy or involuntary twitching.
"With today's CHMP recommendation, Baxter looks forward to offering patients suffering from MMN a licensed treatment option to relieve their symptoms of limb weakness and motor dysfunction," said Professor Hartmut Ehrlich, vice president, global research and development for Baxter's BioScience business. "Baxter remains committed to building upon its long history and proven experience with IVIG therapy to advance treatment in targeted neurological disorders, such as MMN."
The KIOVIG MMN CHMP positive opinion is based on two prospective, open label, investigator initiated clinical efficacy studies in patients with MMN, both showing maintenance of muscle strength and improved functionality. Adverse events were reversible and consistent with those seen in other KIOVIG indications, with no serious adverse events.
"MMN is a debilitating auto-immune disorder that affects individuals' limb strength and motor skills at the prime age of their lives, requiring life-long treatment," said Leonard van den Berg, MD, PhD, Professor in Neurology at the University Medical Center, Utrecht, The Netherlands. "IVIG therapy has long been recognised as recommended therapy for MMN. However, due to the rarity of this disorder the availability of efficacy data and treatment options for MMN remain low despite years of research. An EU-wide approval will mark an important new opportunity for physicians to improve the lives of patients suffering from MMN."
KIOVIG is also the only IVIG treatment offered in a 30 gram vial. This larger vial size offers added convenience of dosing for patients requiring larger doses of IVIG treatment.
About Multifocal Motor Neuropathy (MMN)
MMN is a rare, auto immune-mediated disorder characterized by slowly progressive, asymmetric, distal weakness of one or more limbs, most commonly starting with the arms, leading to significant difficulty with simple manual tasks.1 MMN is caused by disorder/malfunctions in the conduction pathway of motor nerves, limiting transmission of electrical impulses. If left untreated, MMN often progresses to more severe weakness, including muscle atrophy, involuntary twitching and cramps. Almost 80% 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 (3:1). The prevalence is estimated to be 1 per 100,000.
Treatment options for MMN patients are limited. IVIG has been the standard of care for the treatment of MMN for almost 20 years. Available data on the efficacy of IVIg treatment in patients with MMN have been limited due to the rare nature of the condition, small patient populations studied and the inter-individual variability in response rates.
An analysis (Cochrane Review) across all randomized controlled-trials on IVIg treatment in MMN demonstrated a non-significant trend towards improvement in disability after intravenous immunoglobulin compared with placebo, but a significant improvement in muscle strength.2
The treatment of MMN with IVIg is recommended by the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) in their Guideline on Management of Multifocal Motor Neuropathy and the Guideline for the Use of IVIg in the Treatment of Neurological Diseases.
Other Baxter MMN Trials
In 2008, Baxter initiated a Phase 3 clinical trial in the U.S. and Canada for GAMMAGARD Liquid (which is marketed as KIOVIG outside the U.S.) for the treatment of MMN, and upon completion later this year, Baxter intends to seek FDA approval. Baxter has also been granted Orphan Drug Designation for this indication in the United States.
About Autoimmune Disorders
Autoimmune disorders encompass a broad spectrum of human illnesses. Autoimmune diseases are defined as diseases in which the progression from benign autoimmunity to pathogenic autoimmunity occurs. In such cases the immune system mistakenly identifies a person's own tissues (e.g. nerve cells) as foreign and attacks and destroys them. Amongst autoimmune disorders, IVIG products are approved for use in idiopathic thrombocytopenic purpura (ITP), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Guillain Barre syndrome (GBS) and Kawasaki disease.
KIOVIG was approved in Europe in 2006 and has been available in the United States since 2005 (marketed as GAMMAGARD LIQUID™ [Immune Globulin Intravenous (Human)]).
KIOVIG is a human normal immunoglobulin (IVIg), 10% solution indicated for replacement therapy in the treatment of Primary Immunodeficiency Syndromes, hypogammaglobulinemia and recurrent bacterial infections in patients with chronic lymphocytic leukemia and in plateau phase multiple myeloma patients and children and adolescents with congenital AIDS and recurrent bacterial infections.
KIOVIG is also indicated for immunomodulation in idiopathic thrombocytopenic purpura (ITP), Guillain Barré syndrome, Kawasaki Disease; and is indicated for hypogammaglobulinaemia in patients after allogeneic haematopoietic stem cell transplantation. On 24 June 2011, the CHMP issued a positive opinion for extension of the therapeutic indications of KIOVIG to include MMN indication.
On 5 May 2010, the European Commission approved a 30g/300 ml vial size for KIOVIG. The addition of the 30g format can be combined with the 20g, 10g, 5g, 2.5g and 1g vial sizes to meet dosing requirements with fewer vials.
KIOVIG Important Safety Information
Side effects for KIOVIG include the following: very common: headache, pyrexia; common: bronchitis, nasopharyngitis, dizziness, migraine, vertigo, tachycardia, flushing, hypertension, cough, rhinorrhea, diarrhea, nausea, vomiting, pruritis, rash, urticaria, back pain, myalgia, pain in extremity, fatigue, influenza-like illness, infusion site pain, infusion site swelling, rigors, raised body temperature.
See Summary of Product Characteristics for more detail and information on KIOVIG is available on the website of the European Medicines Agency (EMA) http://www.ema.europa.eu/.
Important Safety Information for the United States
KIOVIG is marketed as GAMMAGARD LIQUID™ [Immune Globulin Intravenous (Human)], in the United States.
GAMMAGARD LIQUID is contraindicated in patients with known anaphylactic or severe hypersensitivity responses to Immune Globulin (Human). Patients with severe selective IgA deficiency (IgA < 0.05 g/L) may develop anti-IgA antibodies that can result in a severe anaphylactic reaction. Such patients should only receive intravenous immune globulin with utmost caution and in a setting where supportive care is available for treating life-threatening reactions.
IVIG products have been associated with renal dysfunction, acute renal failure, osmotic nephrosis, and death. While these reports of renal dysfunction and acute renal failure have been associated with the use of many of the licensed IVIG products, those containing sucrose as a stabilizer accounted for a disproportionate share of the total number.
GAMMAGARD LIQUID does not contain sucrose.
GAMMAGARD LIQUID is made from human plasma. Products made from human plasma may carry a risk of transmitting infectious agents, such as viruses, that can cause disease.
The potential risks and benefits of IVIG should be weighed against those of alternative therapies for all patients for whom IVIG administration is being considered.
Please visit www.gammagardliquid.com for full U.S. prescribing information.
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 use of KIOVIG (marketed as GAMMAGARD LIQUID in the United States and Canada) to treat multifocal motor neuropathy (MMN), including expectations with respect to regulatory approvals to be sought for such indication. 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 European Commission; timely submission and approval of anticipated regulatory filings; the successful completion of the Phase 3 clinical trial being conducted at sites across the United States and Canada; 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.
1Nobile-Orazio E. Multifocal motor neuropathy. Journal of Neuroimmunology 115 (2001) 4-18
2van Schaik IN, van den Berg LH, de Haan R, Vermeulen M. Intravenous immunoglobulin for multifocal motor neuropathy. Cochrane Database Syst.Rev. 2005, CD004429