The U.S. Food and Drug Administration (FDA) has approved two Octapharma supplements to Biological License Applications (BLA). The supplement approvals resulted in new product labeling for Octaplas™, Pooled Plasma (Human), Solvent/Detergent (S/D) Treated Solution for Intravenous Infusion, and fibryga®, Fibrinogen (Human) Lyophilized Powder for Reconstitution.
The Octaplas™ labeling now provides the results of two post-marketing studies (LAS-212, LAS-213) evaluating the product’s efficacy, safety and tolerability for newborns through age 20. Octaplas™, an alternative to fresh frozen plasma, was evaluated in 91 pediatric patients up to age 20. There were no hyperfibrinolytic or treatment-related thromboembolic events reported by investigators. Results of these studies support the use of Octaplas™ in critically ill pediatric patients.
“These two studies with Octaplas™ are an important addition to the pediatric literature and provide supportive data for a virus-inactivated plasma option in these patients,” said Cassandra Josephson, MD, Professor of Pathology and Laboratory Medicine and Pediatrics at Emory University School of Medicine in Atlanta, Ga.
The new Octaplas™ prescribing information adds data from the LAS-213 clinical trial to assess the safety and tolerability of the product in the pediatric population. The prospective, open-label, multicenter, single-arm post-marketing study evaluated Octaplas™ in the management of patients who underwent therapeutic plasma exchange (TPE). Patients were dosed based on body weight and doses were adjusted as needed.
A total of 102 TPE procedures were performed in 41 patients aged 2 to 20 years (15 between ages 2 and 12; 13 between ages 12 and 17; and 13 between the ages of 17 and 20). Fourteen participants had immune system disorders; 12 had nervous system disorders; 8 had renal and urinary disorders; 4 had infections and infestations; and 3 had other disorders. No thrombotic or thromboembolic events were found in any study participant. In total, 8 adverse reactions were found in 4 patients. Most adverse reactions (7 of 8) were mild in intensity and were recovered/resolved by the end of the study. No treatment-related serious adverse events were reported. Overall safety was assessed by investigators as excellent for most subjects (>90%) at 24 hours after each TPE throughout the study using prespecified definitions of excellent, good and poor.
An earlier study (LAS-212) published in the Frontiers in Pediatrics, supported the use of Octaplas™ in the management of pediatric patients who require replacement of multiple plasma coagulation factors. 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. Overall safety was assessed by investigators as excellent for all 50 patients.
“We are very pleased to announce the supplemental approvals for both Octaplas™ and fibryga®,” said Octapharma USA President Flemming Nielsen. “Critical care medicine is an important part of Octapharma’s therapeutic focus and we are committed to working with researchers to advance patient treatment options. Providing the resources and expertise to better manage rare disease continues to be one of our main goals.”
For fibryga®, human fibrinogen concentrate, the FDA approved Octapharma’s request to expand the indication for on-demand treatment of acute bleeding episodes to pediatric patients less than 12 years old with congenital fibrinogen deficiency (CFD), also known as Factor 1 deficiency. The expansion was supported by the results of a Phase 3 study recently published in Haemophilia. Researchers observed the efficacy, pharmacokinetics and safety of treatment with fibryga® in 14 pediatric patients with CFD.
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