COUNT ON FABRAZYME.

Choose the proven therapy for Fabry disease with over 17 years of experience.1

MECHANISM OF ACTION

Count on Fabrazyme: Can be used regardless of genotype or disease severity.1

FABRAZYME: For patients with any level of enzyme activity.1

Fabrazyme is a fully functional enzyme, with an amino acid sequence identical to the body’s own native enzyme.1 Fabrazyme binds to a natural receptor on the cell surface, allowing it to be internalized.16 Once inside the cell, Fabrazyme is directly transported to the lysosome.1,16 In the lysosome, Fabrazyme hydrolyzes GL-3, resulting in clearance of accumulated GL-3 in major organs.1

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

Count on FABRAZYME. Count on experience.

FABRAZYME is the longest-studied Fabry disease therapy.17

Fabrazyme has over 17 years of real-world experience
Fabrazyme has been chosen for more than 5000 patients worldwide

CLINICAL STUDIES

COUNT ON FABRAZYME.

FABRAZYME was proven to clear GL-3 in as quickly as 5 months in the capillary endothelium of the kidney, heart, and skin.1

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.1

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.1

FABRAZYME rapidly normalized plasma GL-3 and maintained it for up to 5 years.1,19

  • 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) and heart (6/8) at 4.5 years.1

FABRAZYME WAS EVALUATED FOR OUTCOMES IN CLINICALLY SIGNIFICANT EVENTS.*

A smaller percentage of patients in the FABRAZYME treatment group experienced a clinically significant event.1

28% of Fabrazyme-treated patients versus 42% of placebo-treated patients experienced a clinically significant event

HAD RENAL, CARDIAC, CEREBROVASCULAR EVENTS, OR DEATH*

  • 28% (14 of 51) of Fabrazyme-treated patients versus 42% (13 of 31) of placebo-treated patients experienced a clinically significant event (HR 0.57, 95% CI: 0.27, 1.22, P= 0 .14)
  • The primary efficacy endpoint was the time to first occurrence of a clinically significant event (renal, cardiac, or cerebrovascular event, or death)

The study included patients aged 20 to 72 years (median age: 45), with a baseline median plasma αGAL level of 1.5 nmol/hour/mL (range: 0 to 1.5). Patients included had advanced Fabry disease with mild-to-moderate kidney dysfunction at baseline (median eGFR of 52 mL/min/1.73m2 [range: 25 to 113])1

Study design: A randomized, double-blind, placebo-controlled, multinational, multicenter study of 82 patients (72 males and 10 females) with Fabry disease, all naive to enzyme replacement therapy. Patients were randomly assigned to Fabrazyme 1 mg/kg or placebo every 2 weeks for up to 35 months (median follow up 18.5 months).

FABRAZYME IN PEDIATRIC POPULATIONS.

The overall efficacy and safety profile of FABRAZYME in pediatric patients was consistent with that seen in adults.1

  • This pediatric study evaluated 16 pediatric patients with Fabry disease (14 males, 2 females), aged 8 to 16 years (median 12 years)1
  • In 14 male patients, Fabrazyme normalized plasma GL-3 levels at 24 weeks and sustained levels at 48 weeks1
  • The two female patients’ plasma GL-3 levels remained normal through week 481
  • 12 of the 14 males had GL-3 inclusions at baseline, and all 12 male patients achieved GL-3 inclusion scores of 0 at 24 and 48 weeks of treatment1

FABRAZYME is the ONLY ERT indicated for patients 2 years of age and older.1

  • In an analysis of 24 Fabrazyme-treated pediatric patients with Fabry disease aged 2 to <8 years, plasma GL-3 levels were normalized1
  • At baseline, all male patients had elevated plasma GL-3 (i.e., >7.03 μg/mL)
  • After treatment, plasma GL-3 levels fell within the normal range (i.e., ≤7.03 μg/mL) in 90.9% (20/22), 94.7% (18/19), and 92.3% (12/13) of patients at 6, 12, and 24 months, respectively1

THE RATE OF KIDNEY FUNCTION DECLINE WAS STUDIED IN FABRAZYME-TREATED PATIENTS.

Long-term observational study results show a difference in the mean eGFR slope between the Fabrazyme-treated and untreated-patients.*

1.7 - estimated difference in mean slope of eGFR

ESTIMATED DIFFERENCE IN MEAN SLOPE OF eGFR

  • Estimated difference in mean slope of eGFR was 1.7 (95% CI: 0.5, 3.0) mL/min/1.73m2/year
  • The mean slope of eGFR of the 2 groups were:
    • Fabrazyme-treated group: -1.5 mL/min/1.73m2/year
    • Untreated group: -3.2 mL/min/1.73m2/year
  • The median follow up time was 3 years in the untreated group and 4.5 years in the treated group (maximum follow-up time was 5 years in both groups)

Study design: In a long-term observational study, the rate of decline in renal function (eGFR slope) was assessed in Fabry disease patients aged ≥16 years, treated with Fabrazyme (n=122) and matched to a historical cohort of untreated patients (n=122).

Patient Baseline Characteristics:

  • The median age at Fabrazyme initiation was 35 years. Proportion of male patients=72%. The proportion of patients with a classic phenotype=84%
  • The median baseline eGFR was 93 mL/min/1.73m2
  • The median age at symptom onset was 10 years and median age at diagnosis was 26 years

SAFETY PROFILE

COUNT ON THE WELL-ESTABLISHED SAFETY PROFILE OF FABRAZYME.

The safety of Fabrazyme has been assessed in 4 clinical trials involving 162 patients with over 473 patient-years of experience.1,17

SUMMARY OF ADVERSE REACTIONS1

  • In clinical trials and postmarketing safety experience with Fabrazyme, approximately 1% of patients developed anaphylactic or severe hypersensitivity reactions during Fabrazyme infusion
  • In clinical trials with Fabrazyme, 59% of patients experienced infusion-associated reactions, some of which were severe. Infusion-associated reactions are defined as adverse reactions occurring on the same day as the infusion
  • In patients experiencing infusion-associated reactions, pretreatment with an antipyretic and antihistamine is recommended. Infusion-associated reactions occurred in some patients after receiving pretreatment
  • Higher incidences of hypersensitivity reactions were observed in adult patients with persistent anti-Fabrazyme antibodies and in adult patients with high antibody titer compared to that in antibody negative adult patients
  • 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 if Fabrazyme is administered

SUMMARY OF COMMON ADVERSE REACTIONS IN CLINICAL TRIALS (STUDIES 1 AND 2).1

Occurring in at least 5% of Fabrazyme-treated patients at an incidence >2.5% compared with placebo-treated patients.1

a Includes reports of upper respiratory infection, nasal congestion, sinusitis, respiratory tract congestion, and pharyngitis.

b Includes reports of chills and feeling cold.

c Includes reports of myalgia and muscle spasms.

ADMINISTRATION

COUNT ON OPTIONS.

FABRAZYME can be administered in multiple treatment settings.

Fabrazyme can be administered in physician offices. PHYSICIAN
OFFICES
Fabrazyme can be administered in infusion centers. INFUSION
CENTERS
Fabrazyme can be administered at home. HOME

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 prescribing information. Cambridge, MA. Genzyme Corporation; 2021.
  2. Krasnewich DM, Sidransky E. Chapter 208. In: Goldman L, Schafer AI, eds. Goldman-Cecil Medicine. Amsterdam, Netherlands: Elsevier; 2016;1(25):1399-1403.
  3. Germain DP. Orphanet J Rare Dis. 2010;5:30.
  4. Kolodny EH, Pastores GM. J Am Soc Nephrol. 2002;13(suppl 2):S150-S153.
  5. Desnick RJ Ioannou YA, Eng CM. Chapter 150. In: Valle D, Beaudet Al, Vogelstein B, et al, eds. The Online Metabolic and Molecular Bases of Inherited Disease. New York, NY: McGraw Hill; 2014.
  6. Schiffmann, R; Hughes, DA; Linthorst, GE, et al. Kidney Int. 2017.
  7. Ortiz A, Germain DP, Desnick RJ, et al. Mol Genet Metab. 2018;123(4):416-427.
  8. MacDermot KD, Holmes A, Miners AH. J Med Gen. 2001;38(11):750-760.
  9. MacDermot KD, Holmes A, Miners AH. J Med Gen. 2001;38(11):769-775.
  10. Mehta A. QJ Med. 2002;95:647-653.
  11. Thurberg BL, Politei JM. Hum Pathol. 2012;43(4):610-614.
  12. Aerts JM, Groener JE, Kuiper S, et al. Proc Natl Acad Sci. 2008;(8):2812-2817.
  13. Ellaway C. Transl Pediatr. 2016;5(1):37-42.
  14. Wraith JE, Tylki-Szymanska A, Guffon N, et al. J Pediatr. 2008;152(4):563-570.
  15. Eng CM, Fletcher J, Wilcox WR, et al. J Inherit Metab Dis. 2007;30(2):184-192.
  16. Prabakaran T, Nielsen R, Larsen JV. PLOS ONE. 2011;6(9):e25065.
  17. Data on file. Genzyme Corporation.
  18. Germain DP, Waldek S, Banikazemi M, et al. J Am Soc Nephrol. 2007;18(5):1547-1557.
  19. Eng CM, Guffon N, Wilcox WR, et al. Am J Hum Genet. 2001;68(3):711-722.