Bursting the Bubble!

For many Americans, their only knowledge of Primary Immune Deficiency (PID) disease is the much maligned movie Bubble Boy.  The film was inspired by the true story of David Vetter who suffered from the rare genetic disease severe combined immune deficiency syndrome (SCID). Because of his inability to fight infections, David spent most of his short life living in a series of sterile bubbles.

However, the reality of PID is very different.  First off, it’s not one disease. The World Health Organization recognizes over 150 different forms of PID. Some, such as selective IgA deficiency, are not even that rare, affecting as many as 1:500 of the population.  Furthermore, today, if a baby like David receives a bone marrow transplant within the first 3.5 months of life, the survival rate can be as high as 94 percent.  For many of the other PID diseases, patients can live healthy and productive lives with the help of replacement immune globulin therapy.

Produced from the plasma of thousands of healthy donors, immunoglobulin, is a concentrated mixture of antibodies that provides the patient with an “artificial immune system.”  When first developed in the 1950’s, the treatment was administered by painful intramuscular injections (IMIG).

In the 1970’s, the first intravenous immunoglobulin (IVIG) preparations were developed. These quickly became the standard of therapy, as they were more effective, reducing both the need for antibiotics and hospitalizations for infections.  However, the required monthly infusions could take several hours and were associated with a high incidence of side effects.

In 2006 the FDA approved the first subcutaneous immunoglobulin (SCIg) therapy for PID. Because SCIg can be self-administered, patients can schedule infusions to suit their own lifestyle. The dosage schedule  also allows patients to maintain more consistent immunoglobulin levels and for some, SCIg is also associated with fewer side-effects.

Last week, life got even better for these patients.  CSL Behring announced that the U.S. Food and Drug Administration (FDA) had granted marketing approval for Hizentra™, Immune Globulin Subcutaneous (Human), 20% Liquid. Hizentra is the first 20% concentration SCIg. This concentration enable patients to administer up to 20g of IgG in a single infusion, making it the first once weekly SCIg. It’s also the first SCIg to be stabilized with L-proline, allowing the product to be stored at room temperature.

“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. “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.”

SRxA and Word on Health congratulate CSL Behring on developing a product that will truly improve the lives of patients with PID.