FDA approves CSL Behring's Hizentra for PI

CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Hizentra^TM, Immune Globulin Subcutaneous (Human), 20% Liquid, for treating patients diagnosed with primary immunodeficiency (PI). A once weekly immunoglobulin (Ig) replacement therapy, Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body. Primary immunodeficiencies constitute a group of disorders, usually genetic, that cause a malfunction in all or part of the immune system, thereby rendering the patient unable to fight off infections caused by everyday germs.

“Hizentra is an important new addition to the rapidly growing CSL Behring product portfolio, and further demonstrates our long-standing commitment to the PI and rare disease communities.”

Hizentra is the first 20 percent subcutaneous immunoglobulin (SCIg) approved in the U.S. by the FDA. This high-concentration product is stabilized with L-proline, a naturally-occurring amino acid. L-proline allows Hizentra to be stored at room temperature (up to 25°C [77°F]). Because no refrigeration is necessary, Hizentra is ready to use, offering patients and physicians convenience and portability. Hizentra can be safely self-administered by PI patients under a physician's care.

"As the first SCIg treatment with a 20 percent concentration of immunoglobulin, Hizentra represents an effective, convenient choice of at-home Ig therapy that will allow people with PI to schedule treatment around their busy lives instead of scheduling their lives around treatment,” said Robert Lefebvre, Vice President and General Manager, U.S. Commercial Operations at CSL Behring. “Hizentra is an important new addition to the rapidly growing CSL Behring product portfolio, and further demonstrates our long-standing commitment to the PI and rare disease communities.”

"With its high concentration, Hizentra is a welcome new SCIg treatment option for patients managing primary immunodeficiencies,” said John Sleasman, M.D., Professor and Chief of the Division of Allergy, Immunology and Rheumatology at the University of South Florida College of Medicine, Department of Pediatrics, and one of the investigators on CSL Behring's clinical study of Hizentra. "Hizentra’s ready-to-use attribute will allow patients to infuse the product where and when it suits them, and physicians now have another product to select to best meet the individual needs of their patients.”

For patients with primary immunodeficiencies, immunoglobulin replacement therapy with a product like Hizentra can help treat existing or chronic infections and prevent new infections from occurring. No single treatment works for every type of PI, but infusions of replacement antibodies (immunoglobulins) can help supplement the immune system to prevent infection in nearly three-quarters of PI cases that are due to antibody deficiencies.

Immunoglobulin, or Ig, is a blood component that has become standard immune replacement therapy for most people living with PI, and nearly 70 percent of PI patients receive Ig replacement therapy. Since the 1980s, the first-line therapy for most PI patients has been intravenous immunoglobulin (IVIg), in which immunoglobulin is delivered through a needle into the vein. Many patients, however, cannot easily tolerate intravenous infusions due to serious side effects or poor veins. Hizentra allows patients to use a small, portable pump to self-administer their weekly infusions by injection under the skin (subcutaneous administration).

Hizentra is part of CSL Behring’s Ig franchise, which also includes both the first FDA-approved subcutaneous Ig treatment and the first proline-stabilized IVIg therapy. Hizentra, also stabilized with proline, will be manufactured at CSL Behring’s new state-of-the-art facility, located at its center of excellence for immunoglobulins in Bern, Switzerland. This new manufacturing facility, which uses advanced technologies to ensure product safety and steady supply production, represents CSL Behring’s long-term commitment to global Ig markets.