FDA Approvals Strengthen Octapharma USA Pediatric Critical Care Product Portfolio


FDA Approvals Strengthen Octapharma USA Pediatric Critical Care Product Portfolio

Octaplas™ and fibryga® receive new product labeling following FDA’s approval of BLA supplements to update therapy research

FDA expands fibryga® indication to include treatment of acute bleeding episodes in pediatric congenital fibrinogen deficiency patients under 12 years of age

The U.S. Food and Drug Administration (FDA) has approved two Octapharma supplements to Biological License Applications, strengthening the company’s pediatric critical care product portfolio. 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 (ClinicalTrials.gov: LAS 213, NCT01938378). 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 (ClinicalTrials.gov: LAS-212 NCT02050841) 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.

A recent case report in the Journal of Thrombosis and Haemostasis reported on the treatment of a pediatric congenital afibrinogenemia patient. The patient received fibryga® for long-term prophylaxis after experiencing allergic reactions to other replacement therapies, and for perioperative management during liver transplantation. “Our case report provides further evidence for the safety and tolerability of this human fibrinogen concentrate in a pediatric patient with CFD,” said Fernando F. Corrales-Medina, MD of the University of Miami-Hemophilia Treatment Center.

Additionally, earlier research published in the Journal of Thrombosis and Haemostasis reported on the use of fibryga® for on‐demand treatment of bleeding and surgical prophylaxis in 25 adult and adolescent patients with afibrinogenemia. Fibryga® is not approved for use as prophylactic treatment.

Three research posters on fibryga® will be presented at the virtual American Society of Pediatric Hematology/Oncology (ASPHO) 2021 conference to be held April 20 – 23. The poster titles are:

  • Human Fibrinogen Concentrate for Bleeding Prophylaxis During Surgery in Adult, Adolescent and Pediatric Patients with Congenital Fibrinogen Deficiency: Results from the FORMA-02 and FORMA-04 Clinical Trials
  • Analysis of Fibrinogen Concentrate Pharmacokinetics and Dosing in Adult, Adolescent and Pediatric Patients with Congenital Fibrinogen Deficiency
  • Efficacy and Safety of Human Fibrinogen Concentrate in Pediatric and Adolescent Patients with Congenital Fibrinogen Deficiency: Results from the FORMA-02 and FORMA-04 Clinical Trials

For ASPHO program information, please visit ASPHO.org.

About fibryga®

Fibryga®, Fibrinogen (Human) Lyophilized Powder for Reconstitution, for Intravenous Use, is a human fibrinogen concentrate indicated for the treatment of acute bleeding episodes in adults and children with congenital fibrinogen deficiency, including afibrinogenemia and hypofibrinogenemia. Fibryga® is not indicated for dysfibrinogenemia. Fibryga® is stored as a lyophilized powder at room temperature or refrigerated temperature [+2°C to + 25°C (36°F to 77°F)] for up to 30 months from the date of manufacture. The product can be reconstituted with sterile water within approximately 5 to 10 minutes with the provided Octajet transfer device and particle filter (17-micron).

CONTRAINDICATIONS

Fibryga® is contraindicated in individuals who have manifested severe immediate hypersensitivity reactions, including anaphylaxis, to fibryga® or its components.

WARNINGS AND PRECAUTIONS

  • Monitor patients for early signs of hypersensitivity or allergic reactions. If necessary, discontinue administration and institute appropriate treatment.
  • Thrombotic events have been reported in patients receiving fibryga®. Treatment with human fibrinogen concentrate has been associated with thrombosis at target plasma fibrinogen levels that were below 150 mg/dL. The thrombotic risks may be greater when the target fibrinogen plasma level is 150 mg/dL. Weigh the benefits of administration versus the risks of thrombosis.
  • Fibryga® is made from pooled human plasma. Products made from human plasma may contain infectious agents, e.g., viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

The fibryga® manufacturing process includes a solvent/detergent (S/D) step for virus inactivation, and a nanofiltration step (Planova 20N nanofilter or Pegasus SV4 nanofilter) for virus removal. For complete prescribing information, please visit fibrygausa.com.

FDA Approvals Strengthen Octapharma USA Pediatric Critical Care Product Portfolio

EUA200090 states, “Saliva specimens must be collected, transported and stored using the Spectrum Solutions LLC SDNA-1000 Saliva Collection Device.”

Spectrum Solutions, LLC today announced the FDA Emergency Use Authorization (EUA) approval for the immediate use of saliva in COVID-19 testing exclusively using the Spectrum SDNA-1000 saliva collection device which has been proven to protect and preserve RNA used for the detection of infection.

The issued EUA for saliva as a viable biosample in COVID-19 testing came from the result of a detailed saliva vs. swab study strategically looking for an answer to the devastating swab and media supply shortages felt not only nationwide but globally. The study conclusion uncovered a novel, and technically robust testing alternative using saliva collected with Spectrum’s SDNA-1000 saliva collection device. These results fast-tracked EUA approval for RUCDR Infinite Biologics – Rutgers Clinical Genomics Laboratory and its Perkin Elmer nucleic acid extraction and ThermoFisher TaqPath SARS-CoV-2 automated processes using the Spectrum Solutions SDNA-1000 device.

“Using saliva to test for COVID-19 overcomes many of the challenges the nation faces that are inherent to current testing methods including supply shortages and risks to exposure for healthcare professionals,” said Stephen Fanning, CEO of Spectrum Solutions. “Now, under a medical professional’s direction, the saliva collection can be self-administered by individuals who may be in quarantine or self-isolation, removing the need to be in close contact with medical staff.”

“The Spectrum Solutions SDNA-1000 Saliva Collection Device was chosen to collect, transport and store saliva specimens for the TaqPath SARS-CoV-2 Assay proof-point study given RUCDR’s experience with Spectrum products across a wide range of applications currently employed,” said Dr. Andrew Brooks, Chief Operating Officer at RUCDR Infinite Biologics and Professor of Genetics at Rutgers University. “The preservation solution was a main focus of the study to ensure that the virus would be inactivated to the point that we would be able to maintain and stabilize the RNA transcripts for sensitive and specific QPCR analysis in order to determine whether a person has the virus. The device’s intuitive ease-of-use to facilitate minimally supervised or in most cases complete self-collection is a tremendous advance to current COVID-19 sample collection strategies. Together, these benefits will significantly add to expanding access to critical testing needs.”

FDA Approvals Strengthen Octapharma USA Pediatric Critical Care Product Portfolio

Medlab Asia and Asia Health, a free-to-attend virtual event brought by Informa Markets and Impact Exhibition Management Co., kicks off today Tuesday 20 October with a panel discussion on pandemic management. This year’s event builds on the success of ASEAN’s biggest multi-disciplinary conference for the healthcare and medical laboratory industry.

The three day virtual exhibition will bring together regional healthcare professionals to meet and do business online. Participants may explore hundreds of innovative products from businesses seeking new dealers and distributors – including the latest from leading global players such as Abbott, Euroimmunn, LG, Randox and Snibe – and also set up virtual meetings to discuss new business opportunities.

The event will also empower attendees with advanced knowledge and networking skills to improve patient service and care. More than 50 healthcare, laboratory and business sessions will be available through keynotes, on-demand broadcasts and virtual panel discussions on topics that include COVID-19 Control & Treatment, Imaging, Doing Business in Thailand and Immunology.

Attendees will earn up to 21 CME, 15 CMTE and 7 CNEU points through hearing from more than 100 world renowned experts in healthcare. Speakers include Dr Tanarak Plipat, Deputy Director General, Department of Disease Control, Ministry of Public Health, Thailand;  Prof Heungsup Sung, Chairperson, COVID-19 Diagnosis Test Management Committee, Korea Centers for Disease Control and Prevention, South Korea; Dr Bayu Teja Muliawan, Head of Planning Republic of Indonesia, Ministry of Health, Indonesia; and Joseph Kalaivanar, CEO, GotMyCoach, Singapore.

“We are excited to be introducing Medlab Asia & Asia Health this year as a virtual edition, enabling participants to interact and do business with the wider healthcare and medical laboratory community, while also learning through a compelling line-up of accredited webinars – all from the comfort of their own home or office,” says Tom Coleman, Exhibitions Director. 

For more information, visit www.medlabasia/asiahealth.com

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