Octapharma USA: FDA Approves New Octaplas Product Label

The U.S. Food and Drug Administration (FDA) has approved a revised product label for Octapharma USA’s Octaplas, Pooled Plasma (Human), Solvent/Detergent Treated Solution for Intravenous Infusion. The new labeling provides the results of a post-marketing study on Octaplas that demonstrates the product’s safety, efficacy and tolerability in treating critically ill pediatric patients who require replacement of multiple coagulation factors.

“The results of this study provide clinical evidence supporting the use of Octaplas in critically ill pediatric patients with acquired deficiencies who require replacement of multiple coagulation factors due to liver disease or who are undergoing cardiac or liver transplant surgery,” said Octapharma USA President Flemming Nielsen. “The results of the present study and those of previous studies comparing Octaplas™ with fresh frozen plasma support its use in this patient population.”¹

Study Details
The prospective, open-label, multicenter, single arm, post-marketing study assessed 50 pediatric patients age 16 years and under (37 neonates/infants, less than age 2; and 13 children/adolescents, age 2 to 16). Study participants included 40 cardiac surgery patients, 5 liver transplant/dysfunction patients, 4 sepsis-related coagulopathy patients and 1 patient with hypoxic encephalopathy. For complete trial details, please visit ClinicalTrials.gov; clinical trial identifier: NCT02050841.

Patients were dosed based on body weight and doses were adjusted as needed. There were no hyperfibrinolytic or treatment-related thromboembolic events reported by investigators. Overall safety was assessed by investigators as “excellent” for all 50 patients. Hemostatic parameters as measured by international normalized ratio (INR), prothrombin time (PT), activated partial thromboplastin time (aPTT), and thromboelastography (TEG) were within expected ranges following use of Octaplas.¹

The study results demonstrating the safety of Octaplas in the management of critically ill pediatric patients were presented at the Society of Cardiovascular Anesthesiologists Annual Meeting.

Background
Correction of an abnormal coagulation profile is the most common indication for plasma transfusions.² Clinical practice guidelines recommend plasma transfusion in children and neonates with an abnormal coagulation profile and clinically significant bleeding or prior to invasive procedures with a high bleeding risk.³

Several types of plasma products are available for transfusion, including fresh frozen plasma (FFP) or solvent/detergent (S/D)-treated, pooled plasma.⁴ Octaplas is a S/D treated, virus inactivated, blood group-specific human plasma prepared from pooling multiple single donor FFP units resulting in a standardized composition with significantly lower variability in coagulation factor content compared to standard FFP.⁵ In comparison to FFP, Octaplas is practically cell free, and contains significantly lower residual platelet concentrations and negligible amounts of microparticles due to its manufacturing process.⁶ Octaplas offers a safety benefit as a result of the pathogen inactivation and removal of cells or cell fragments during the manufacturing process.

^1. Data on file, Octapharma USA, 2019.
^2. Labarinas, S., Arni, D., Karam, O.: Plasma in the PICU. Annals of Intensive Care. 3, 16 (2013).
^3. New, H. V., Berryman, J., Bolton-Maggs, et al.: Guidelines on transfusion for fetuses, neonates and older children. Br. J. Haematol. 175, 784–828 (2016).
^4. Camazine, M. N., Karam, O., Colvin, R., et al.: Outcomes Related to the Use of Frozen Plasma or Pooled Solvent/Detergent-Treated Plasma in Critically Ill Children. Pediatric critical care medicine a journal of the Society of Critical Care Medicine and the World Federation of Pediatric Intensive and Critical Care Societies. 18, e215-e223 (2017).
^5. Heger, A., Romisch, J., Svae, T.-E.: A biochemical comparison of a pharmaceutically licensed coagulation active plasma (Octaplas) with a universally applicable development product (Uniplas) and single-donor FFPs subjected to methylene-blue dye and white-light treatment. Transfus. Apher. Sci. 35, 223–233 (2006).
^6. Spinella, P. C., Frazier, E., Pidcoke, et al.: All plasma products are not created equal. J. Trauma Acute Care Surg. 78, S18-25 (2015).