Once-weekly immunoglobulin under skin injection Hizentra for primary immunodeficiency approved in US: CSL Behring

Hizentra, a 20 percent concentration of human immunoglobulin to be administered subcutaneously for treating patients diagnosed with primary immunodeficiency (PI), has been granted approval in US.

Once weekly immunoglobulin (Ig) replacement therapy, Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body, announced the developer of the drug CSL Behring in a press release.

Hizentra is claimed to be the first 20 percent subcutaneous immunoglobulin (SCIg) approved in the U.S.

Hizentra is derived from human plasma.This high-concentration immunoglobulin 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,

“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,” stated Robert Lefebvre, vice president and general manager, U.S. Commercial Operations at CSL Behring.

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.

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 primary immunodeficiencies patients has been intravenous immunoglobulin (IVIg), in which immunoglobulin is delivered through a needle into the vein.

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 primary immunodeficiencies cases that are due to antibody deficiencies.

Many patients, however, cannot easily tolerate intravenous infusions due to serious side effects or poor veins.

Hizentra has a small, portable pump to self-administer 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 will be manufactured at CSL Behring’s new state-of-the-art facility, located at its center of excellence for immunoglobulins in Bern, Switzerland.

What is primary immunodeficiency?

Primary immunodeficiencies are 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.

Nearly 100 types of primary immunodeficiencies exist. For individuals with primary immunodeficiencies, many of them children, infections may not improve as expected with usual treatments and may keep returning.

As a result, patients may face repeated rounds of antibiotics or hospitalization for treatment. Repeated infections can lead to organ damage, which over time can become life-threatening. Some infections, such as meningitis, may even result in death.

Primary immunodeficiencies affect an estimated 10 million people worldwide, and the incidence is estimated to be 1 in 10,000.

Due to the X-linked inheritance in many primary immunodeficiencies syndromes, more males are affected than females.

CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company headquartered in Melbourne, Australia.

CSL Behring is a leader in the plasma protein therapeutics industry. The company manufactures and markets a range of plasma-derived and recombinant therapies worldwide.

CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies and inherited respiratory disease.

The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma.