PRIVIGEN® (Immune Globulin Intravenous (Human), 10% Liquid) Granted Orphan-Drug Designation for the Investigational Treatment of Systemic Sclerosis (SSc)

KING OF PRUSSIA, Pa., Feb. 11, 2020 /PRNewswire/ -- Global biotherapeutics leader CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted Privigen^® (Immune Globulin Intravenous (Human),10% Liquid)  orphan-drug designation as an investigational therapy in the treatment of Systemic Sclerosis (SSc). SSc is a chronic and potentially life-threatening autoimmune disorder characterized by a build-up of scar tissue (fibrosis) in the skin and other organs.¹ It affects approximately 100,000 people in the U.S.² There are currently no FDA-approved,  disease-modifying treatments to stop or reverse the overall course of the disease.

The FDA Office of Orphan Products Development (OOPD) grants orphan-drug designation to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 U.S. patients. The designation qualifies companies that invest in research and development of these medications for a range of incentives, including fee waivers, tax credits and the potential for marketing exclusivity upon approval.

"CSL Behring is driven by our promise to develop and deliver innovative therapies for patients with the highest unmet need," said Mittie Doyle, Vice President, Research and Development, Immunology and Neurology Therapeutic Area at CSL Behring. "Receiving orphan drug designation for Privigen as an investigational SSc therapy is an important milestone in our quest to address the devastating impact of systemic sclerosis."

Privigen is currently approved in the U.S. to treat patients with primary immunodeficiency (PI), chronic inflammatory demyelinating polyneuropathy (CIDP) and chronic immune thrombocytopenic purpura (ITP).  CSL Behring recently initiated a Phase 2 clinical trial to evaluate the safety and efficacy of Privigen in the treatment of adults with SSc, and in October 2019 received Fast Track Designation for the clinical development of Privigen for the treatment of serious pulmonary, skin, and musculoskeletal manifestations resulting from this condition. The FDA confers Fast Track designation to aid and expedite the development and review of drugs that show promise in treating a serious or life-threatening disease and address an unmet medical need.

About Systemic Sclerosis
Systemic Sclerosis (SSc) is a rare, chronic, and potentially life-threatening autoimmune rheumatic disease characterized by the buildup of scar tissue or fibrosis resulting in hardening, thickening and tightening of the skin. In SSc, fibrosis can also affect internal organs, such as the esophagus, heart, lungs, and kidneys, often leading to their impairment or failure.¹ SSc affects approximately 100,000 people in the United States, with females being four times more likely to develop the condition than males. Other factors, such as race and ethnicity, may also influence the risk of getting SSc, the age of onset and the pattern and severity of internal organ involvement.^1-2

About Privigen®
Privigen® is the first and only 10 percent, ready-to-use, liquid IVIg stabilized with proline, a naturally occurring amino acid. Privigen was first approved in 2007 in U.S. for the treatment of patients with primary immunodeficiency (PI) and chronic immune thrombocytopenic purpura (ITP) in patients age 15 years and older. Since September 2017, Privigen has also been indicated for the maintenance treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) in adults and is available in more than 60 countries around the world to treat these and other rare diseases. For more information about Privigen, including the U.S. Prescribing Information, visit www.privigen.com.

Important Safety Information

Immune Globulin Intravenous (Human), 10% Liquid, Privigen®, is indicated for the treatment of:

• Primary humoral immunodeficiency (PI)
• Chronic immune thrombocytopenic purpura (ITP) in patients age 15 years and older
• Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults
• Limitation of use: maintenance therapy in CIDP has not been studied for periods longer than 6 months. Individualize duration of treatment beyond 6 months based on patient response.

WARNING: THROMBOSIS, RENAL DYSFUNCTION AND ACUTE RENAL FAILURE

• Thrombosis may occur with immune globulin products, including Privigen. 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.
• Renal dysfunction, acute renal failure, osmotic nephrosis, and death may occur with immune globulin intravenous (IGIV) products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products that contain sucrose. Privigen does not contain sucrose.
• For patients at risk of thrombosis, renal dysfunction or renal failure, administer Privigen 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.

See full prescribing information for complete boxed warning.

Privigen is contraindicated in patients with history of anaphylactic or severe systemic reaction to human immune globulin, in patients with hyperprolinemia, and in IgA-deficient patients with antibodies to IgA and a history of hypersensitivity.

In patients at risk of developing acute renal failure, monitor urine output and renal function, including blood urea nitrogen and serum creatinine.

Hyperproteinemia, increased serum viscosity, or hyponatremia can occur with Privigen. Infrequently, aseptic meningitis syndrome (AMS) may occur—especially with high doses or rapid infusion.

Hemolysis, either intravascular or due to enhanced red blood cell sequestration, may occur. Risk factors include non-O blood group and high doses. Closely monitor patients for hemolysis and hemolytic anemia.

During and shortly following Privigen infusion, elevations of systolic and diastolic blood pressure (including cases of hypertensive urgency) have been observed. These elevations resolved or significantly improved within hours with oral anti-hypertensive therapy or observation alone. Check patients for a history of hypertension and monitor blood pressure during this period.

Consider relative risks and benefits before prescribing high-dose regimen for chronic ITP and CIDP in patients at increased risk of thrombosis, hemolysis, acute kidney injury or volume overload. Monitor patients for pulmonary adverse reactions (transfusion-related acute lung injury [TRALI]).

Privigen is derived from human plasma. 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.

In clinical studies of patients with PI, the most common adverse reactions to Privigen, observed in >5% of subjects, were headache, fatigue, nausea, chills, vomiting, back pain, pain, elevated body temperature, abdominal pain, diarrhea, cough, stomach discomfort, chest pain, joint swelling/effusion, influenza-like illness, pharyngolaryngeal pain, urticaria, and dizziness. Serious adverse reactions were hypersensitivity, chills, fatigue, dizziness, and increased body temperature.

In clinical studies of patients being treated for chronic ITP, the most common adverse reactions, seen in >5% of subjects, were laboratory findings consistent with hemolysis, headache, elevated body temperature, anemia, nausea, and vomiting. A serious adverse reaction was aseptic meningitis syndrome.

In clinical studies of patients being treated for CIDP, the most common reactions, observed in >5% of subjects, were headache, asthenia, hypertension, nausea, pain in extremity, hemolysis, influenza-like illness, leukopenia, and rash. Serious adverse reactions were hemolysis, exacerbation of CIDP, acute rash, increased diastolic blood pressure, hypersensitivity, pulmonary embolism, respiratory failure, and migraine.

Treatment with Privigen might interfere with a patient's response to live virus vaccines and could lead to misinterpretation of serologic testing. In patients over 65 and those at risk of renal insufficiency, do not exceed recommended dose and infuse at the minimum rate practicable.

Full Privigen prescribing information, including the complete boxed warning, can be found at www.privigen.com.

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.

1. NIH health conditions: https://ghr.nlm.nih.gov/condition/systemic-scleroderma. Accessed 2.10.2020
2. Scleroderma Foundation: Disease Reference - What is Scleroderma? www.scleroderma.org; Accessed 2.10.2020

SOURCE CSL Behring