Efficacy & Safety Data

XYNTHA—Proven efficacy and an established safety profile1-4

Bleed Control and Prevention Prophylaxis Inhibitor Data Adverse Reactions

Proven bleed control in PTPs

In the pivotal trial including adults and adolescents

of bleeds were controlled successfully on demand with 1 or 2 infusions

  • Subjects rated first infusion outcomes as excellent or good (71%), moderate (24%), or no response (3%); 3% of first infusion outcomes were not rated
  • In PTPs, 187 bleeding episodes were treated with 282 infusions (median dose: 30.6 IU/kg)

Rating scale:
Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. Moderate: Probable or slight improvement starting within 8 hours following the infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. No response: No improvement at all between infusions or during the 24-hour interval following an infusion, or condition worsens.

Study design
Open-label study of PTPs (mean age, 27.7 years; range, 12-60 years) with severe or moderately severe hemophilia A (FVIII:C ≤2%; ≥150 previous exposure days) given XYNTHA 3 times per week (30 ± 5 IU/kg; N=94) and on demand (investigator-determined dose; n=53 [187 bleeding episodes]). Results reported here for 3-times-per-week administration are based on 89 patients accruing ≥50 exposure days to XYNTHA.

In adolescents​​​​​​​

of bleeds were controlled successfully with 1 or 2 on-demand infusions

  • Subjects rated first infusion outcomes as excellent or good (58%) or moderate (36%); 6% of first infusion outcomes were not rated
  • A total of 66 bleeding episodes were treated with on-demand infusions of XYNTHA

Results based on 18 PTPs 12 to <17 years of age with ≥150 previous exposure days with baseline FVIII activity of ≤2%. Of the 18 subjects <17 years of age who received at least 1 dose of XYNTHA, 10 subjects had bleeding episodes during the study.
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Rating scale:
Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. Moderate: Probable or slight improvement starting within 8 hours following the infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. No response: No improvement at all between infusions or during the 24-hour interval following an infusion, or condition worsens.

Study design
The open-label study of PTPs included 18 adolescents, 12 to <17 years of age who received XYNTHA for on-demand and follow-up treatment. Results reported here for 10 patients (66 bleeding episodes). The median dose/infusion was 47 IU/kg.

In children

of bleeds were controlled successfully with 1 or 2 infusions

  • Subjects rated first infusion outcomes as excellent or good (94%) or moderate (5%)
  • A total of 562 bleeding episodes were treated with on-demand infusions of XYNTHA

Results based on 50 PTPs <16 years of age with ≥20 previous exposure days with baseline FVIII activity of ≤2%. Of the 50 subjects <16 years of age who received at least 1 dose of XYNTHA, 38 had bleeding episodes during the study.

Rating scale:
Excellent: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered. Good: Definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. Moderate: Probable or slight improvement starting within 8 hours following the infusion, with at least 1 additional infusion administered for complete resolution of the bleeding episode. No response: No improvement at all between infusions or during the 24-hour interval following an infusion, or condition worsens.

Study design
Children (n=50) <16 years of age with severe or moderately severe hemophilia A (FVIII:C ≤2% and with ≥20 prior exposure days) received XYNTHA for on-demand and follow-up treatment. Results reported here for 38 patients (562 bleeding episodes). The median dose/infusion was 28 IU/kg. In comparison to the pharmacokinetic parameters reported in adults, children have shorter half-lives, larger volumes of distribution, and lower recovery of factor VIII after XYNTHA administration. Larger or more frequent doses may be required to account for the observed differences in pharmacokinetic parameters.

During and after surgery

of bleed control ratings were excellent or good

  • Perioperative control: 100% of bleed control ratings were excellent (72%) or good (28%) during and up to 1 hour after surgery
  • Postoperative control: 100% of bleed control ratings were excellent (92%) or good (8%) at the end of the postoperative period
  • Intraoperative blood loss was reported as normal or absent for all subjects. Thirteen subjects had blood loss in the postoperative period. This blood loss was rated normal for 10 cases and abnormal for 3 cases (hemorrhage following surgical trauma to epigastric artery, 800-mL blood loss after hip replacement surgery, and after elbow synovectomy where blood loss could not be measured by investigator)

Perioperative management: Open-label study (n=30) for surgical prophylaxis in PTPs with severe or moderately severe hemophilia A (FVIII:C ≤2%) undergoing major surgical procedures. Results reported here for 25 patients who received XYNTHA replacement therapy for at least 6 days post surgery.

Rating scale:
Efficacy responses were assessed as follows: Excellent: Achieved hemostasis comparable to that expected after similar surgery in a patient without hemophilia. Good: Prolonged time to hemostasis, with somewhat increased bleeding compared with that expected after similar surgery in a patient without hemophilia. Moderate: Obviously delayed hemostasis, but manageable with additional infusions. None: No hemostatic response.

Physicians/surgeons rated the efficacy of XYNTHA in supporting the patient through the surgical procedure using the Surgical Hemostasis Efficacy rating scale at the end of surgery and at the end of the initial postoperative period for 25 patients: phase 3, open-label, multicenter, prospective study performed at a total of 13 study sites.
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References:
  1. XYNTHA SOLOFUSE. Prescribing information. Pfizer Inc.; 2020. 
  2. Recht M, Nemes L, Matysiak M, et al. Clinical evaluation of moroctocog alfa (AF-CC), a new generation of B-domain deleted recombinant factor VIII (BDDrFVIII) for treatment of haemophilia A: demonstration of safety, efficacy, and pharmacokinetic equivalence to full-length recombinant factor VIII. Haemophilia. 2009;15(4):869-880. doi:10.1111/j.1365-2516.2009.02027.x
  3. Windyga J, Rusen L, Gruppo R, et al. BDDrFVIII (moroctocog alfa [AF-CC]) for surgical haemostasis in patients with haemophilia A: results of a pivotal study. Haemophilia. 2010;16(5):731-739. doi:10.1111/j.1365-2516.2010.02239.x
  4. Data on file. Pfizer Inc., New York, NY.

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  • Do not use in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product or its components, including hamster proteins.
  • Allergic-type hypersensitivity reactions, including anaphylaxis, are possible with XYNTHA. Inform patients of the early signs or symptoms of hypersensitivity reactions (including hives, generalized urticaria, chest tightness, wheezing, and hypotension) and anaphylaxis. Discontinue XYNTHA if hypersensitivity symptoms occur and administer appropriate emergency treatment. XYNTHA contains trace amounts of hamster proteins. Patients may develop hypersensitivity to these proteins.
  • Inhibitors have been reported following administration of XYNTHA. Monitor patients for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests.
  • Clinical response to XYNTHA may vary. If bleeding is not controlled with the recommended dose of factor, determine the plasma level and administer a dose of XYNTHA sufficient to achieve clinical response. If the factor level does not increase or there is no response, suspect an inhibitor and perform appropriate testing.
  • Across all studies, the most common adverse reactions (≥10%) with XYNTHA in previously treated adult and pediatric patients were headache (24% of subjects), arthralgia (23%), fever (23%), and cough (12%). Other adverse reactions reported in ≥5% of subjects were diarrhea, vomiting, and weakness.
  • XYNTHA is an injectable medicine administered by intravenous (IV) infusion. Patients should be advised that local irritation may occur when infusing XYNTHA after reconstitution in XYNTHA® SOLOFUSE®.

XYNTHA® Antihemophilic Factor (Recombinant) is indicated in adults and children with hemophilia A for on-demand treatment and control of bleeding episodes, for perioperative management, and for routine prophylaxis to reduce the frequency of bleeding episodes. 

XYNTHA is not indicated in patients with von Willebrand’s disease. 

Please see full Prescribing Information for XYNTHA and XYNTHA SOLOFUSE.

Indication

XYNTHA® Antihemophilic Factor (Recombinant) is indicated in adults and children with hemophilia A for on-demand treatment and control of bleeding episodes, for perioperative management, and for routine prophylaxis to reduce the frequency of bleeding episodes. 

XYNTHA is not indicated in patients with von Willebrand’s disease. 

Please see full Prescribing Information for XYNTHA and XYNTHA SOLOFUSE.