Japan's Ministry of Health, Labour and Welfare Approves CSL Behring Immunoglobulins to Treat Patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Hizentra® [human normal immunoglobulin, 20%, subcutaneous] is the first and only subcutaneous immunoglobulin approved for the maintenance treatment of CIDP in Japan, based on findings from the longest controlled clinical CIDP study

Privigen® [human normal immunoglobulin, 10% liquid, intravenous], ready-to-use therapy now approved in Japan for acute and maintenance treatment of CIDP

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CSL Behring

Mar 26, 2019, 12:21 ET

TOKYO, March 26, 2019 /PRNewswire/ -- Global biotherapeutics leader CSL Behring announced today that Japan's Ministry of Health, Labour and Welfare has approved two of its immunoglobulin therapies for the treatment of patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Hizentra, previously approved to treat primary immunodeficiency, is now the first and only subcutaneous immunoglobulin (SCIG) approved for maintenance therapy to treat CIDP in Japan. Privigen, an intravenous immunoglobulin (IVIG), is now approved for both acute and maintenance therapy of CIDP in Japan.

The approval for Hizentra is based on the results of PATH (Polyneuropathy And Treatment with Hizentra) and its extension study, which comprise the longest CIDP research period to date and included a cohort of Japanese patients. The approval for Privigen is based on results from two Phase III clinical studies that focused on the use of immunoglobulin therapy for treating CIDP: the PATH study and the PRIMA (Privigen Impact on Mobility and Autonomy) study.

The studies showed that 73 percent (PATH) and 61 percent (PRIMA) of patients responded to Privigen as measured by their adjusted score on the Inflammatory Neuropathy Cause and Treatment (INCAT) scale, which measures the ability to walk and perform tasks. This improvement continued and was maintained during maintenance treatment of Privigen in the PATH study. Further results from the PATH study demonstrated that after switching from IVIG, the percentage of patients experiencing CIDP relapse or withdrawal for any other reason during SCIG treatment was significantly lower with Hizentra (38.6 percent on low-dose Hizentra [0.2 g/kg weekly]; 32.8 percent on high-dose Hizentra [0.4 g/kg weekly]; p values = 0.007 and <0.001 respectively) than with placebo (63.2 percent). In addition, approximately three times as many patients preferred subcutaneous treatment over intravenous treatment.

CIDP is a chronically progressive rare autoimmune disorder that affects the peripheral nerves and may cause permanent nerve damage. The myelin sheath, or the protective covering of the nerves, is damaged, which may result in numbness or tingling, muscle weakness, fatigue and other symptoms which worsen over time.

"CIDP treatment aims to achieve long-term remission and maintain motor and sensory function, with minimal adverse effects," said Prof. Satoshi Kuwabara, Department of Neurology, Chiba University. "However, currently available treatment may not be sufficiently effective for some patients, and many CIDP patients continue to suffer disabling symptoms due to the disease and resulting lifestyle changes. We expect that novel treatment options, such as Hizentra that may be self-administered and Privigen that has been proven to have high evidence for its efficacy and safety in a large clinical trial, will improve long-term prognosis and quality of life of CIDP patients."

The effects of CIDP lead to significant activity limitations and a decreased quality of life. Approximately 30 percent of CIDP patients will progress to wheelchair dependence if not treated. Until now, the only immunoglobulin therapy available to treat CIDP in Japan was intravenously administered.

William Mezzanotte, Executive Vice President, Research & Development, CSL Limited reinforced a commitment to the CIDP patient community saying, "CSL Behring has dedicated years to studying CIDP to bring patients effective treatment options that fit their lifestyle. We are proud to offer Japanese patients with CIDP a balanced portfolio of immunoglobulin therapies with both Hizentra, a convenient option with proven efficacy and the flexibility and freedom to self-infuse, and Privigen, with its long-proven track record of safety."

About CIDP
In CIDP, a rare autoimmune disorder that affects the peripheral nerves (those outside the brain and spinal cord), the myelin sheath, the protective covering of the nerves, is damaged. CIDP effects can worsen over time, leading to significant activity limitations and a decreased quality of life. CIDP can occur at any age but peak prevalence is between 40 and 60 years of age and is more common in men than in women. Approximately 30 percent of CIDP patients will progress to wheelchair dependence if not treated. It is estimated that the global prevalence for CIDP is 1.6 to 8.9 patients per 100,000 adults.

About Hizentra^®Hizentra [Human normal immunoglobulin (SCIG)], the first 20 percent SCIG developed for subcutaneous use, is available in more than 45 countries to treat certain immune deficiencies and as a maintenance treatment for CIDP. Hizentra, the world's most prescribed SCIG, has a proven track record of safety, efficacy and tolerability and has over 6 million exposures worldwide since 2010.

About Privigen^®Privigen [Human normal immunoglobulin (IVIG)] is a 10 percent, ready to use, liquid IVIG stabilized with proline. A naturally occurring amino acid, proline has been shown to reduce IgG aggregation and dimer formation. It is available in more than 60 countries around the world and is used as replacement therapy for patients with primary and secondary immunodeficiencies, and as an immunomodulatory therapy for patients with immune thrombocytopenia (ITP), Kawasaki disease, and neurological disorders such as CIDP, multifocal motor neuropathy (MMN) and Guillain-Barré syndrome.

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; USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 22,000 people, and delivers its life-saving therapies to people in more than 60 countries. For inspiring stories about the promise of biotechnology, visit Vita at CSLBehring.com/Vita and follow us on Twitter.com/CSLBehring.

SOURCE CSL Behring