Hoboken-based Octapharma USA Announces FDA Approval of Octagam10%

On Jul 16, 2014

Hoboken-based Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved Octagam10% [Immune Globulin Intravenous (Human) 10% (100 mg/mL) Liquid Preparation] for the treatment of adults with chronic Immune Thrombocytopenic Purpura (ITP), a platelet disorder that can result in easy or excessive bruising and bleeding.

The FDA approval marks a significant expansion of Octapharma’s U.S. immune globulin therapy portfolio. Octapharma USA is a subsidiary of Octapharma AG, a global human protein products manufacturer, and since 2004 has been marketing Octagam® 5% [Immune Globulin Intravenous (Human) 5% (50 mg/mL) Liquid Preparation] to treat Primary Humoral Immunodeficiency (PI).

“The FDA approval of Octagam10% represents a significant Octapharma milestone, but, more importantly, opens up new treatment options for patients,” said Octapharma USA President Flemming Nielsen. “Octagam10% has demonstrated safety and efficacy in a pivotal clinical trial. Octapharma looks forward to providing the product in the U.S., further advancing our vision of providing patients with therapies to treat life-threatening conditions.”

Octagam10% is expected to be available in the U.S. in September 2014. Further details will be announced shortly.

The approval of Octagam10% is based on the results of an Octapharma-sponsored clinical trial to evaluate the safety and efficacy of Octagam10% in 66 patients (age: 17-88 years) with chronic ITP (ClinicalTrials.gov Identifier NCT00426270).

In the October 2010 issue of the journal Hematology, Robak et al published the article, “Efficacy and safety of a new intravenous immunoglobulin 10% formulation (Octagam10%) 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 7 days of dosing), which was significantly higher than the predicted responder rate of 70%. In this study, there were no unexpected tolerability issues even at the maximum infusion rate of 0.12 mL/kg/minute (720 mg/kg/hour). The researchers further reported that 78% of patients with chronic ITP, who had bleeding at baseline, reported no bleeding 7 days after treatment.

The most common treatment-related adverse events observed with Octagam10% treatment during the clinical trial were: headache, fever, and increased heart rate. The most serious adverse event observed with Octagam10% treatment during the clinical trial was a moderate headache.