Octapharma USA Announces FDA Approval for 2nd Octagam® 10% Manufacturing Site, Expanding Product Availability

Immune Therapy Production Facilities FDA-Licensed in Both Vienna and Stockholm

HOBOKEN, N.J.--(BUSINESS WIRE)--Octapharma USA today announced that the U.S. Food and Drug Administration (FDA) has approved the company’s manufacturing facility in Vienna, Austria for the production of Octagam 10% [Immune Globulin Intravenous (Human) 10% (100 mg/mL) Liquid Preparation], which became available in the U.S. during October 2014. Octagam 10% for the U.S. market can now be manufactured at FDA-licensed facilities in both Stockholm, Sweden and Vienna.

“The FDA approval of Octapharma’s Vienna manufacturing site for Octagam 10% is great news for patients, as it will help facilitate product availability and enhances production flexibility,” said Flemming Nielsen, President of Octapharma USA. “Octapharma owns six manufacturing facilities internationally, which all utilize the latest technology and strict quality control processes. Octapharma is committed to providing access to life-saving products, while continuing to focus on patient tolerability and safety.”

Octapharma USA is a subsidiary of Octapharma AG, a global human protein products manufacturer, and has since 2004 been marketing Octagam 5% [Immune Globulin Intravenous (Human) 5% (50 mg/mL) Liquid Preparation] to treat Primary Humoral Immunodeficiency (PI). Octagam 5% is also manufactured at the FDA-licensed manufacturing facilities in Stockholm and Vienna.

Octapharma’s additional state-of-the-art manufacturing facilities are located in Lingolsheim, France; Dessau and Springe, Germany; and Mexico City, Mexico. Octapharma’s production facilities perform plasma fractionation and purification to produce biopharmaceuticals, and/or product labeling, packaging, storage, and distribution. Research and development takes place in Berlin, Frankfurt and Heidelberg, Germany and Lachen, Switzerland.

In July 2014, the FDA approved Octagam 10% for the treatment of adults with chronic Immune Thrombocytopenic Purpura (ITP), a platelet disorder that can result in easy or excessive bruising and bleeding. In a pivotal study in adults with chronic ITP, Octagam 10% delivered a rapid and sustained clinical outcome. The infusion rate was up to 12 mg/kg/min (720 mg /kg/hour), without compromising tolerability.^{1, 2}

An Octapharma-sponsored clinical trial evaluated the safety and efficacy of Octagam 10% in 66 patients (age: 17-88 years) with chronic ITP (ClinicalTrials.gov Identifier NCT00426270).^{1, 2} The study results were published in the journal Hematology, entitled “Efficacy and safety of a new intravenous immunoglobulin 10% formulation (Octagam 10%) in patients with immune thrombocytopenia.” The study observed that 82% of patients with chronic ITP attained the primary efficacy endpoint of clinical response (platelet count ≥50×10⁹/L within seven days of dosing), which was significantly higher than the predicted responder rate of 70%. The researchers further reported that 78% of patients with bleeding at baseline reported no bleeding on day seven and 84% of patients reported a decrease in bleeding severity by day seven. The patients were evaluated during a 21-day study period and at a 63-day follow-up assessment.²

In this study, there were no unexpected tolerability issues even at the maximum infusion rate. Among initial study phase patients, 92% were titrated to the initial maximum infusion rate of 6 mg/kg/min (360 mg/kg/hour) and 60% of subsequent patients were titrated to the amended maximum infusion rate of 12 mg/kg/min (720 mg/kg/hour).^{1, 2}

The most common treatment-related adverse events observed with Octagam 10% treatment during the clinical trial were: headache, fever, and increased heart rate. The most serious adverse event observed with Octagam 10% treatment during the clinical trial was a moderate headache. ^{1, 2}

About Octagam 10% liquid

Octagam 10% [Immune Globulin Intravenous (Human) 10% (100 mg/mL) Liquid Preparation] is indicated for chronic ITP in adults to rapidly raise platelet counts to control or prevent bleeding. Octagam 10% is a solvent/detergent (S/D) treated, sterile preparation of highly purified immunoglobulin G (IgG) derived from large pools of human plasma. Octagam 10% is a solution for infusion to be administered intravenously.

WARNING: THROMBOSIS, RENAL DYSFUNCTION and ACUTE RENAL FAILURE

Please see full prescribing information for complete boxed warning.

• Thrombosis may occur with immune globulin intravenous (IGIV) products, including Octagam 5% and Octagam 10% liquid. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling central vascular catheters, hyperviscosity, and cardiovascular risk factors.
• Renal dysfunction, acute renal failure, osmotic nephropathy, and death may occur with the administration of Immune Globulin Intravenous (Human) (IGIV) products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products containing sucrose. Octagam 5% and Octagam 10% liquid do not contain sucrose.
• For patients at risk of thrombosis, renal dysfunction or renal failure, administer Octagam 5% and Octagam 10% liquid at the minimum 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.

CONTRAINDICATIONS

Octagam 10% is contraindicated in patients who have a history of severe systemic hypersensitivity reactions, such as anaphylaxis, to human immunoglobulin. Octagam 10% contains trace amounts of IgA (average 106 µg/mL in a 10% solution). It is contraindicated in IgA-deficient patients with antibodies against IgA and history of hypersensitivity.

WARNINGS AND PRECAUTIONS

IgA-deficient patients with antibodies against IgA are at greater risk of developing severe hypersensitivity and anaphylactic reactions to Octagam 10%. Epinephrine should be available immediately to treat any severe acute hypersensitivity reactions. Monitor renal function, including blood urea nitrogen and serum creatinine, and urine output in patients at risk of developing acute renal failure. Falsely elevated blood glucose readings may occur during and after the infusion of Octagam 10% with testing by some glucometers and test strip systems. Hyperproteinemia, increased serum osmolarity and hyponatremia may occur in patients receiving Octagam 10%. Thrombotic events may occur. Monitor patients with known risk factors for thrombotic events; consider baseline assessment of blood viscosity for those at risk of hyperviscosity. Aseptic Meningitis Syndrome may occur in patients receiving Octagam 10%, especially with high doses or rapid infusion. Hemolytic anemia can develop subsequent to IGIV treatment. Monitor patients for hemolysis and hemolytic anemia. Monitor patients for pulmonary adverse reactions (transfusion-related acute lung injury (TRALI)). Octagam 10% is made from human plasma and may contain infectious agents, e.g. viruses and, theoretically, the Creutzfeldt-Jakob disease agent.

For full prescribing information, please visit www.octagamus.net.

About the Octapharma Group

Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein products manufacturers in the world and has been committed to patient care and medical innovation since 1983. Its core business is the development and production of human proteins from human plasma and human cell-lines. Octapharma employs approximately 6,000 people worldwide to support the treatment of patients in over 100 countries with products across the following therapeutic areas: Hematology (coagulation disorders), Immunotherapy (immune disorders) and Critical Care. The company’s American subsidiary, Octapharma USA, is located in Hoboken, N.J. Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration (FDA), providing a high level of production flexibility. For more information, please visit www.octapharmausa.com, www.octagamus.net, www.octaplasus.com or www.wilateusa.com.

REFERENCES

1. Octagam 10% [Immune Globulin Intravenous (Human)] Liquid Preparation. Complete Prescribing Information. Octapharma. Hoboken, New Jersey, USA.

2. Robak, T, Mainau, C, Pyringer, B et al. Efficacy and safety of a new intravenous immunoglobulin 10% formulation (Octagam 10%) in patients with immune thrombocytopenia. Hematology 2010; 15:351-359.

3. Octapharma. Data on File. 2015

Forward-looking Statements

This news release contains forward-looking statements, which include known and unknown risks, uncertainties, and other factors not under the company’s control. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. These factors include results of current or pending research and development activities and action by the FDA or other regulatory authorities.

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