First and only subcutaneous immunoglobulin (SCIg) approved for maintenance therapy in CIDP qualifies for marketing exclusivity
KING OF PRUSSIA, Pa.– December 9, 2019 – Global biotherapeutics leader CSL Behring announced today that Hizentra received orphan-drug exclusivity from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impairment. CIDP is a rare autoimmune disorder that affects the peripheral nerves and has the potential to cause significant disability.
Hizentra was approved by the FDA in March 2018 for the treatment of adults with CIDP to prevent relapse of neuromuscular disability and impairment. It is the first and only 20 percent subcutaneous immunoglobulin (SCIg) approved for this indication. The approval was based on data from the Phase III PATH (Polyneuropathy And Treatment with Hizentra) study, the largest controlled clinical study in CIDP patients to date. In the PATH trial, patients taking Hizentra relapsed or withdrew less often than those taking placebo. Patients in the study also maintained their grip strength as well as upper and lower body strength.
The FDA’s decision regarding the orphan drug status of Hizentra provides CSL Behring a seven- year period of U.S. marketing exclusivity for Hizentra in the maintenance treatment of CIDP with SCIg. The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. and qualifies incentives for companies that invest in research and development of these medications.
“Orphan drug exclusivity is a significant milestone for the CSL Behring team committed to delivering Hizentra and improving the lives of patients diagnosed with CIDP,” said Bob Lojewski, Senior Vice President and General Manager, North America, CSL Behring. “We are proud to be the only company to offer an innovative portfolio of subcutaneous and intravenous immunoglobulin therapies for CIDP. Driven by our promise, we will continue our work to fulfill the needs of patients with serious conditions like CIDP.”
Registered in more than 60 countries, Hizentra is the world's most prescribed SCIg and has a proven track record of safety, efficacy, and tolerability, with more than 6.7 million exposures worldwide since 2010. Hizentra was first approved by the U.S. FDA in March 2010 for the treatment of patients with primary immunodeficiency (PI). For more information about Hizentra, including the U.S. prescribing information, visit www.hizentra.com.
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 will 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.
Important Hizentra Safety Information for the U.S.
Hizentra®, Immune Globulin Subcutaneous (Human), 20% Liquid, is indicated for:
Treatment of primary immunodeficiency (PI) in adults and pediatric patients 2 years and older.
Maintenance therapy in adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to prevent relapse of neuromuscular disability and impairment.
Limitation of use: Maintenance therapy in CIDP has been systematically studied for 6 months and for a further 12 months in a follow-up study. Continued maintenance beyond these periods should be individualized based on patient response and need for continued therapy.
For subcutaneous infusion only.
WARNING: Thrombosis may occur with immune globulin products, including Hizentra. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors.
For patients at risk of thrombosis, administer Hizentra at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.
Hizentra is contraindicated in patients with a history of anaphylactic or severe systemic reaction to human immune globulin (Ig) or components of Hizentra (eg, polysorbate 80), as well as in patients with immunoglobulin A deficiency with antibodies against IgA and a history of hypersensitivity. Because Hizentra contains L-proline as stabilizer, use in patients with hyperprolinemia is contraindicated.
IgA-deficient patients with anti-IgA antibodies are at greater risk of severe hypersensitivity and anaphylactic reactions. Thrombosis may occur following treatment with Ig products, including Hizentra.
Monitor patients for aseptic meningitis syndrome (AMS), which may occur following treatment with Ig products, including Hizentra. In patients at risk of acute renal failure, monitor renal function, including blood urea nitrogen, serum creatinine and urine output. In addition, monitor patients for clinical signs of hemolysis or pulmonary adverse reactions (eg, transfusion-related acute lung injury [TRALI]).
Hizentra is derived 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 adverse reactions (observed in ≥5% of study subjects) were local infusion-site reactions, as well as headache, diarrhea, fatigue, back pain, nausea, extremity pain, cough, upper respiratory tract infection, rash, pruritus, vomiting, upper abdominal pain, migraine, arthralgia, pain, fall, and nasopharyngitis.
The passive transfer of antibodies can interfere with response to live virus vaccines and lead to misinterpretation of serologic test results.
Please see full prescribing information for Hizentra.
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 25,000 people worldwide, 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.
Dana Lynch, Immunology and Neurology Communications