KING OF PRUSSIA, Pa. – April 29, 2021 – Global biotherapeutics leader CSL Behring today announced that the U.S. Food and Drug Administration (FDA) has approved a label update for Hizentra® (Immune Globulin Subcutaneous [Human] 20% Liquid) for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). The approval, based on data from the PATH (Polyneuropathy And Treatment with Hizentra) Extension study, simplifies dosage adjustments between two safe and effective doses proven to prevent CIDP relapse - without the need for intravenous immune globulin treatment in the event of a relapse on the low dose. The PATH Extension study was a multicenter, open-label extension study to the Phase III PATH study that evaluated the long-term safety and efficacy of Hizentra 0.2 g/kg and 0.4 g/kg weekly doses in the maintenance treatment of CIDP.
“This label update and data show that physicians can confidently adjust their patient’s subcutaneous immune globulin treatment,” said Arie Katz, Senior Medical Director at CSL Behring. “Providing consistency in a proven treatment can lessen some of the burden that patients living with CIDP may face.”
Hizentra is the most prescribed self-infused subcutaneous immune globulin (SCIg) treatment and the first and only SCIg treatment approved for CIDP, allowing CIDP patients the flexibility to self-infuse their immune globulin treatment at home. In 2020, CSL Behring introduced the first and only pre-filled syringes as a simple, convenient and ready-to-use option.
“At CSL Behring, we are dedicated to improving the lives of people with rare and serious diseases and we are fully committed to our promise of innovating treatments that lessen the burden patients and physicians may face in managing CIDP,” said Bernadine Koziara, Vice President, Marketing, CSL Behring. “This approval will add another great benefit for physicians and patients, providing greater flexibility and a more streamlined treatment approach to deliver the best possible care for CIDP.”
About the PATH Extension Study
The 48-week, open-label, prospective extension study to PATH enrolled 82 patients, with 62 patients starting on 0.4 g/kg weekly infusion of Hizentra and 20 patients starting on 0.2 g/kg weekly. If clinically stable, patients on 0.4 g/kg were switched to 0.2 g/kg after 24 weeks. If relapse occurred while on the 0.2 g/kg dose, 0.4 g/kg was either initiated or reinitiated. While most patients remained relapse-free while on either dose of Hizentra, of the 72 patients who received 0.4 g/kg at any point during the extension study, 90% remained relapse-free. Results demonstrated that the 0.4 g/kg dose had a higher likelihood of preventing relapse than the 0.2 g/kg dose.
The PATH study was a Phase III clinical trial designed to demonstrate the efficacy, safety and tolerability of two different doses of Hizentra, compared with placebo, in the maintenance treatment of CIDP patients previously treated with intravenous immune globulin. The PATH study was the largest ever CIDP trial.
CIDP is a rare autoimmune disorder that affects the peripheral nerves (those outside the brain and spinal cord) and damages the protective covering of the nerves known as the myelin sheath. This may result in numbness or tingling, muscle weakness, fatigue and other symptoms. CIDP effects can worsen over time, leading to significant activity limitations and a decreased quality of life. CIDP can occur at any age and is more common in men than in women. The GBS|CIDP Foundation estimates that approximately 30 percent of CIDP patients progress to wheelchair dependence if not treated. The Foundation also estimates that the incidence of CIDP in the U.S. is as high as two in 100,000 people each year, with the accumulation of cases over time resulting in prevalence as high as nine in 100,000 in some areas.
Registered in more than 60 countries, Hizentra is the world's most prescribed subcutaneous immunoglobulin for primary immunodeficiency (PI), with more than 9.3 million exposures worldwide since 2010. It has a proven track record of safety, efficacy, and tolerability. Hizentra was first approved by the U.S. FDA in March 2010 for the treatment of patients with PI and was approved in March 2018 for the treatment of adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) to prevent relapse of neuromuscular disability and impairment. For more information about Hizentra, including the U.S. prescribing information, visit www.hizentra.com.
IMPORTANT SAFETY INFORMATION
Hizentra®, Immune Globulin Subcutaneous (Human), 20% Liquid, is a prescription medicine used to treat:
- Primary immune deficiency (PI) in patients 2 years and older
- Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults
WARNING: Thrombosis (blood clots) can occur with immune globulin products, including Hizentra. Risk factors can include: advanced age, prolonged immobilization, a history of blood clotting or hyperviscosity (blood thickness), use of estrogens, installed vascular catheters, and cardiovascular risk factors.
If you are at high risk of blood clots, your doctor will prescribe Hizentra at the minimum dose and infusion rate practicable and will monitor for signs of clotting events and hyperviscosity. Always drink sufficient fluids before infusing Hizentra.
See your doctor for a full explanation, and the full prescribing information for complete boxed warning.
Treatment with Hizentra might not be possible if your doctor determines you have hyperprolinemia (too much proline in the blood), or are IgA-deficient with antibodies to IgA and a history of hypersensitivity. Tell your doctor if you have previously had a severe allergic reaction (including anaphylaxis) to the administration of human immune globulin. Tell your doctor right away or go to the emergency room if you have hives, trouble breathing, wheezing, dizziness, or fainting. These could be signs of a bad allergic reaction.
Inform your doctor of any medications you are taking, as well as any medical conditions you may have had, especially if you have a history of diseases related to the heart or blood vessels, or have been immobile for some time. Inform your physician if you are pregnant or nursing, or plan to become pregnant.
Infuse Hizentra under your skin only; do not inject into a blood vessel. Self-administer Hizentra only after having been taught to do so by your doctor or other healthcare professional, and having received dosing instructions for treating your condition.
Immediately report to your physician any of the following symptoms, which could be signs of serious adverse reactions to Hizentra:
- Reduced urination, sudden weight gain, or swelling in your legs (possible signs of a kidney problem).
- Pain and/or swelling or discoloration of an arm or leg, unexplained shortness of breath, chest pain or discomfort that worsens on deep breathing, unexplained rapid pulse, or numbness/weakness on one side of the body (possible signs of a blood clot).
- Bad headache with nausea; vomiting; stiff neck; fever; and sensitivity to light (possible signs of meningitis).
- Brown or red urine; rapid heart rate; yellowing of the skin or eyes; chest pains or breathing trouble; fever over 100°F (possible symptoms of other conditions that require prompt treatment).
Hizentra is made from human blood. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent and its variant (vCJD), cannot be completely eliminated.
The most common side effects in the clinical trials for Hizentra include redness, swelling, itching, and/or bruising at the infusion site; headache; chest, joint or back pain; diarrhea; tiredness; cough; rash; itching; fever, nausea, and vomiting. These are not the only side effects possible. Tell your doctor about any side effect that bothers you or does not go away.
Before receiving any vaccine, tell immunizing physician if you have had recent therapy with Hizentra, as effectiveness of the vaccine could be compromised.
Please see full prescribing information for Hizentra, including boxed warning and the patient product information.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.
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, the company develops and delivers innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company’s products are also used in cardiac surgery, 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 27,000 people worldwide, and delivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit Vita at CSLBehring.com/Vita and follow us on Twitter.com/CSLBehring.
Jennifer Purdue, External Communications Manager, CSL Behring
Phone: (610) 306-9355