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COUNT ON FABRAZYME: FOR YOUR PATIENTS WITH FABRY DISEASE.

Fabrazyme has more than 15 years of real-world experience and has been chosen for more than 4500 patients worldwide.1

MECHANISM OF ACTION

Count on Fabrazyme: Can be used regardless of genotype or disease severity.2

Fabrazyme: For patients with any level of enzyme activity.2

Fabrazyme is a fully functional enzyme, with an amino acid sequence identical to the body’s own native enzyme.2 Fabrazyme binds to a natural receptor on the cell surface, allowing it to be internalized.3 Once inside the cell, Fabrazyme is directly transported to the lysosome.3 In the lysosome, Fabrazyme hydrolyzes GL-3, resulting in clearance of accumulated GL-3 in major organs.2

WATCH THIS VIDEO TO LEARN HOW FABRAZYME WORKS TO CLEAR GL-3.

CLINICAL STUDIES

COUNT ON FABRAZYME.

FABRAZYME cleared GL-3 as quickly as 5 months in the capillary endothelium of the kidney, heart, and skin.2

Study 1: At 5 months, a GL-3 inclusion score of 0 was achieved in the capillary endothelium of the: Kidney 20 (69%) Fabrazyme patients compared with 0 (0%) placebo patients; Heart: 21 (72%) Fabrazyme patients compared with 1 (3%) placebo patient; Skin: 29 (100%) Fabrazyme patients compared with 1 (3%) placebo patient.2

Study 1 open-label extension: At 6 months, the majority of patients treated with Fabrazyme had a GL-3 inclusion score of 0 in the capillary endothelium of the kidney, heart, and skin.2,4

COUNT ON FABRAZYME.

FABRAZYME rapidly normalized plasma GL-3 and maintained it for up to 5 years.2,5

  • Similar long-term responses were seen in a majority of patients, with sustained GL-3 clearance in the capillary endothelium of the kidney (8/8), heart (6/8), and skin (31/36) at 4.5 years.2
  • The reduction of GL-3 inclusions suggests that Fabrazyme may ameliorate disease expression; however, the relationship of GL-3 inclusion reduction to specific clinical manifestations of Fabry disease has not been established.2

Fabrazyme also clears GL-3 in pediatric populations.2

The overall efficacy and safety profile of Fabrazyme in pediatric patients ≥8 years was consistent with that seen in adults.2*

  • In 12 male patients, Fabrazyme achieved GL-3 inclusion scores of 0 in the capillary endothelium of the skin at 24 weeks and 48 weeks. Two male patients had no measurable GL-3 inclusions at baseline.2
  • In 14 male patients, Fabrazyme normalized plasma GL-3 levels at 24 weeks and sustained levels at 48 weeks.2
  • The overall efficacy and safety profile of Fabrazyme in pediatric patients was consistent with that seen in adults. IgG seroconversion in pediatric patients was associated with prolonged half-life of Fabrazyme, a phenomenon rarely observed in adult patients.2

SAFETY PROFILE

COUNT ON THE WELL-ESTABLISHED SAFETY PROFILE OF FABRAZYME.1, 2

The safety of Fabrazyme has been assessed in 4 clinical trials involving 162 patients with over 473 patient-years of experience.1, 2

SUMMARY OF ADVERSE REACTIONS2

  • Approximately 1% of patients in clinical trials and postmarketing safety experience developed life-threatening anaphylactic or severe allergic reactions.
  • In clinical trials, 59% of patients experienced infusion-associated reactions during Fabrazyme treatment, some of which were severe.
  • Over time, infusion-associated reactions tended to decline during clinical trials; however, they may still occur despite extended duration of treatment.
  • A standard pretreatment approach with an antipyretic and antihistamine is recommended for patients experiencing infusion-associated reactions; however, in clinical trials, infusion reactions occurred in some patients after receiving pretreatment.
  • Patients with advanced Fabry disease may have compromised cardiac function, which may predispose them to a higher risk of severe complications from infusion-associated reactions; these patients should be monitored closely during Fabrazyme administration.
  • Re-administration of Fabrazyme to patients who have previously experienced severe or serious allergic reactions to Fabrazyme should be done only after careful consideration of the risks and benefits of continued treatment, and only under the direct supervision of qualified personnel and with appropriate medical support measures readily available.

SUMMARY OF COMMON ADVERSE REACTIONS IN CLINICAL TRIALS.2

Occurring in at least 5% of Fabrazyme-treated patients at an incidence ≥2.5% compared with placebo-treated patients.2

ADMINISTRATION

COUNT ON OPTIONS.

FABRAZYME can be administered in multiple treatment settings.

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OFFICES
INFUSION
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  • Choice of treatment setting is at the discretion of the physician.
  • Because of the potential for severe infusion-associated reactions, appropriate medical support measures should be readily available.

DOSING AND ADMINISTRATION

The recommended dosage of Fabrazyme is 1 mg/kg body weight infused every 2 weeks as an intravenous (IV) infusion.1

For step-by-step instructions on how to calculate dose, preparation for use, storage and handling, and administering Fabrazyme, download the Fabrazyme Preparation & Administration Info Sheet.

WATCH THIS TUTORIAL VIDEO TO LEARN MORE ABOUT ADMINISTERING FABRAZYME.

References:
  1. Fabrazyme data on file.
  2. Fabrazyme prescribing Information. Cambridge, MA. Genzyme Corporation.
  3. Prabakaran T, Nielsen R, Larsen JV, ea al. PLoS ONE. 2011;6(9):e25065.
  4. Germain DP, Waldek S, Banikazemi M, et al. J Am Soc Nephrol. 2007;18(5):1547-1557.
  5. Eng CM, Banikazemi M, Gordon RE, et al. Am J Hum Genet 2001;68(3):711-722.