FDA Accepts CSL Behring's Supplemental Biologics License Application for Hizentra® Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Indication

KING OF PRUSSIA, Pa., July 19, 2017 /PRNewswire/ -- Global biotherapeutics leader CSL Behring today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's supplemental Biologics License Application (BLA) for Hizentra^® [Immune globulin subcutaneous (Human) 20% liquid] for the treatment for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impairment. CIDP is a rare immune-mediated disorder of the peripheral nerves and the effects can worsen over time.

"We remain committed to CIDP patients and their families and the review of this application is another step towards delivering on our promise to them." said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited. "We're also excited about the possibility of adding a CIDP indication for our industry-leading portfolio of immunoglobulin therapies."  

The application was based on data from the largest-ever randomized CIDP trial, PATH (Polyneuropathy And Treatment with Hizentra^®).  The clinical trial was completed in March and was designed to demonstrate the efficacy, safety and tolerability of two different doses of CSL Behring's subcutaneous immunoglobulin (SCIG), Hizentra^®, compared with placebo, in the maintenance treatment of CIDP patients previously treated with intravenous immunoglobulin (IVIG).

Hizentra^®, the no. 1 prescribed immunoglobulin therapy in treating primary immunodeficiencies (PI), the most prescribed SCIG worldwide, and the only 20 percent SCIG designed with the natural stabilizer L-proline, was self-administered by patients and care givers throughout the study.  Subjects were allowed to use dose volumes up to 50 mL/site and infusion rates of up to 35 mL/hour, to provide them with greater flexibility and autonomy to infuse when and where they choose. A long-term open label extension study is ongoing and is expected to be completed later this year.

About Hizentra^®

Hizentra^® (Immune Globulin Subcutaneous [Human]), the first 20 percent SCIG developed for subcutaneous use, is registered in over 46 countries and approved in North America, Europe and Japan. Hizentra^®, the world's most prescribed SCIG, has a proven track record of safety, efficacy and tolerability and has over 3.6 million exposures worldwide since 2010. In the United States, Hizentra^® is indicated for the treatment of patients with primary immunodeficiency. In all 29 European/European Economic Area member states and Japan, Hizentra^® is authorized for treating patients diagnosed with PI as well as secondary immunodeficiencies.

For country specific indication information, visit:

• Switzerland: https://compendium.ch/mpro/mnr/22484/html/de (German) or https://compendium.ch/mpro/mnr/22484/html/fr (French)
• Europe: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002127/human_med_001440.jsp&mid=WC0b01ac058001d124
• United States: http://www.hizentra.com/Professional/Prescribing-Information.aspx
• Canada: http://labeling.cslbehring.ca/PM/CA/Hizentra/EN/Hizentra-Product-Monograph.pdf
• Australia: http://www.csl.com.au/productfinder/hizentraau

Important Safety Information

Immune Globulin Subcutaneous (Human), Hizentra^®, is indicated as replacement therapy for patients with primary humoral immunodeficiency (PI), age 2 and older. This includes but is not limited to the humoral immune defect in congenital agammaglobulinemia, common variable immunodeficiency, X-linked agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies.

WARNING: THROMBOSIS - Thrombosis may occur with immune globulin products, including Hizentra^®. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors.

For patients at risk of thrombosis, administer Hizentra^® at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity. See full prescribing information for complete boxed warning.

Hizentra^® is contraindicated in patients with a history of anaphylactic or severe systemic reaction to human immune globulin preparations or components of Hizentra^®, such as polysorbate 80. Because it contains the stabilizer L-proline, Hizentra^® is contraindicated in patients with hyperprolinemia. Hizentra^® is also contraindicated in patients with immunoglobulin A deficiency who have antibodies against IgA and a history of hypersensitivity.

Hizentra^® should be administered subcutaneously only. Do not administer intravenously.

IgA-deficient patients with anti-IgA antibodies may be at greater risk of developing potentially severe hypersensitivity and anaphylactic reactions with administration of Hizentra^®. If hypersensitivity occurs or anaphylactic reactions are suspected, discontinue administration immediately and treat as medically appropriate.

Monitor patients for aseptic meningitis syndrome (AMS), which has been reported with SCIg. In patients at risk of acute renal failure, monitor renal function, including blood urea nitrogen, serum creatinine and urine output. Also monitor patients for clinical signs of hemolysis or transfusion-related acute lung injury (TRALI).

Hizentra^® is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.

The most common adverse reactions (observed in 5% or more of study subjects receiving Hizentra^®) were local reactions (ie, swelling, redness, heat, pain, and itching at the injection site), headache, diarrhea, fatigue, back pain, nausea, extremity pain, cough, rash, pruritus, vomiting, upper abdominal pain, migraine and pain.

Ig administration can transiently impair the efficacy of live attenuated virus vaccines, such as measles, mumps and rubella. It can also lead to misinterpretation of serologic testing.

Please see full prescribing information for Hizentra^® including boxed warning.

About CIDP
CIDP is a rare disorder of the peripheral nerves (those outside the brain and spinal cord). The condition is immune-mediated and the effects can worsen over time. The protective covering of the nerves is damaged, which may cause numbness or tingling, muscle weakness, fatigue and other symptoms. CIDP can occur at any age and is more common in men than in women. If left untreated, approximately 30 percent of CIDP patients will progress to wheelchair dependence.

For more information about the PATH study, visit http://www.clinicaltrials.gov and search with identifier: NCT01545076.

About CSL Behring
CSL Behring is a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX: CSL), headquartered in Melbourne, Australia, employs nearly 20,000 people, delivering its life-saving therapies to people in more than 60 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.

SOURCE CSL Behring

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