Agalsidase beta: A review of its use in the management of Fabry disease

Drugs

Volumn 67, Issue 3, 2007, Pages 435-456

  Keating, Gillian M ^a,b   Simpson, Dene ^a,b  

^a ADIS INTERNATIONAL LIMITED   (New Zealand)

^b WOLTERS KLUWER HEALTH   (United States)

Author keywords

Agalsidase beta; Fabry disease; Pharmacodynamics; Pharmacokinetics; Therapeutic use; Tolerability

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GALACTOSIDASE; AMIODARONE; CHLOROQUINE; GENTAMICIN; GLOBOTRIAOSYLCERAMIDE; MONOBENZONE; PLACEBO;

BRAIN BLOOD FLOW; CARDIOVASCULAR RISK; CHILL; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; DRUG BLOOD LEVEL; DRUG EFFICACY; DRUG HALF LIFE; DRUG MECHANISM; DRUG TOLERABILITY; DRUG WITHDRAWAL; FABRY DISEASE; FEVER; FOLLOW UP; HEADACHE; HUMAN; HYDROLYSIS; HYPERTENSION; HYPOTENSION; IMMUNOGENICITY; INJECTION SITE REACTION; KIDNEY FUNCTION; MYALGIA; NERVE FUNCTION; NONHUMAN; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; REVIEW; RIGOR; SEROCONVERSION; THROAT DISEASE; URTICARIA;

EID: 33947316741     PISSN: 00126667     EISSN: 00126667     Source Type: Journal    
DOI: 10.2165/00003495-200767030-00007     Document Type: Review

References (66)

1. Desnick RJ, Brady R, Barranger J, et al. Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med 2003 Feb 18; 138 (4): 338-46
2. Hopkin RJ, Bissler J, Grabowski GA. Comparative evaluation of α-galactosidase A infusions for treatment of Fabry disease. Genet Med 2003 May/Jun 30; 5 (3): 144-53
3. MacDermot KD, Holmes A, Miners AH. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females. J Med Genet 2001; 38: 769-807
4. MacDermot KD, Holmes A, Miners AH. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males. J Med Genet 2001; 38: 750-60
5. Lee K, Jin X, Zhang K, et al. A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease. Glycobiology 2003 Apr; 13 (4): 305-13
6. Genzyme Corporation. Fabrazyme® (agalsidase beta) for intravenous infusion: prescribing information [online]. Available from URL: http://www.fabrazyme.com [Accessed 2005 Oct 5]
7. European Medicines Agency. Fabrazyme: summary of product characteristics [online]. Available from URL: http://www.emea.eu.int [Accessed 2007 Jan 3]
8. Ioannou YA, Zeidner KM, Gordon RE, et al. Fabry disease: preclinical studies demonstrate the effectiveness of α-galactosidase A replacement in enzyme-deficient mice. Am J Hum Genet 2001; 68: 14-25
9. Blom D, Speijer D, Linthorst GE, et al. Recombinant enzyme therapy for Fabry disease: absence of editing of human α-galactosidase A mRNA. Am J Hum Genet 2003 Jan; 72 (1): 23-31
10. Eng CM, Banikazemi M, Gordon RE, et al. A phase 1/2 clinical trial of enzyme replacement in Fabry disease: pharmacokinetic, substrate clearance, and safety studies. Am J Hum Genet 2001 Mar; 68 (3): 711-22
11. Eng CM, Guffon N, Wilcox WR, et al. Safety and efficacy of recombinant human α-galactosidase A replacement therapy in Fabry's disease. N Engl J Med 2001 Jul 5; 345 (1): 9-16
12. Kosch M, Koch H-G, Oliveira JP, et al. Enzyme replacement therapy administered during hemodialysis in patients with Fabry disease. Kidney Int 2004; 66 (3): 1279-82
13. European Medicines Agency. Fabrazyme: scientific discussion [online]. Available from URL: http://www.emea.eu.int [Accessed 2005 Oct 5]
14. Siamopoulos KC. Fabry disease: kidney involvement and enzyme replacement therapy. Kidney Int 2004 Feb; 65 (2): 744-53
15. Banikazemi M, Bultas J, Waldek S, et al. Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med 2007 Jan 16; 146 (2): 77-86
16. Wilcox WR, Banikazemi M, Guffon N, et al. Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am J Hum Genet 2004 Jul; 75 (1): 65-74
17. Germain DP, Waldek S, Banikazemi M, et al. Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease. J Am Soc Nephrol. In press
18. Thurberg BL, Rennke H, Colvin RB, et al. Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy. Kidney Int 2002 Dec; 62 (6): 1933-46
19. Thurberg BL, Byers HR, Granter SR, et al. Monitoring the 3-year efficacy of enzyme replacement therapy in Fabry disease by repeated skin biopsies. J Invest Dermatol 2004 Apr; 122 (4): 900-8
20. Wraith J, on behalf of the AGAL-016 Study Investigators. Agalsidase beta (recombinant human α-galactosidase A) therapy in paediatric patients with Fabry disease [abstract]. 10th International Congress of Inborn Errors of Metabolism; 2006 Sep 12-16; Chiba
21. Eto Y, Ohashi T, Utsunomiya Y, et al. Enzyme replacement therapy in Japanese Fabry disease patients: the results of a phase 2 bridging study. J Inherit Metab Dis 2005; 28 (4): 575-83
22. Pisani A, Spinelli L, Sabbatini M, et al. Enzyme replacement therapy in Fabry disease patients undergoing dialysis: effects on quality of life and organ involvement. Am J Kidney Dis 2005 Jul; 46 (1): 120-7
23. Beer M, Weidemann F, Breunig F, et al. Impact of enzyme replacement therapy on cardiac morphology and function and late enhancement in Fabry's cardiomyopathy. Am J Cardiol 2006; 97: 1515-8
24. Spinelli L, Pisani A, Sabbatini M, et al. Enzyme replacement therapy with agalsidase β improves cardiac involvement in Fabry's disease. Clin Genet 2004 Aug; 66 (2): 158-65
25. Weidemann F, Breunig F, Beer M, et al. Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease: a prospective strain rate imaging study. Circulation 2003 Sep 16; 108 (11): 1299-301
26. Kalliokoski RJ, Kantola I, Kalliokoski KK, et al. The effect of 12-month enzyme replacement therapy on myocardial perfusion in patients with Fabry disease. J Inherit Metab Dis 2006; 29 (1): 112-8
27. Mignani R, Panichi V, Giudicissi A, et al. Enzyme replacement therapy with agalsidase beta in kidney transplant patients with Fabry disease: a pilot study. Kidney Int 2004 Apr; 65 (4): 1381-5
28. Breunig F, Beer M, Knoll A, et al. Kidney function, myocardial mass and late enhancement in 17 patients with Fabry disease after 1 year of enzyme replacement therapy [abstract no. MP142]. 41st Congress of the European Renal Association and the European Dialysis and Transplant Association; 2004 May 15-18; Lisbon
29. Guffon N. Stabilisation of kidney function in a group of 15 patients diagnosed with Fabry disease and treated with Fabrazyme® for a year [abstract no. W272]. World Congress on Nephrology; 2003 Jun 8-12; Berlin
30. Hilz MJ, Brys M, Marthol H, et al. Enzyme replacement therapy improves function of C-, Aδ-, and Aβ-nerve fibers in Fabry neuropathy. Neurology 2004 Apr 13; 62 (7): 1066-72
31. Hilz MJ, Marthol H, Brys M, et al. Treatment with recombinant human alpha-galactosidase A improves cerebral blood flow velocity in Fabry patients [abstract no. P29]. 4th European Round Table on Fabry Disease; 2003 Oct 17-18; Munich
32. Hilz MJ, Szczepanska H, Tutaj M, et al. Enzyme replacement therapy: stability of neurophysiological and autonomic function in Fabry patients within the infusion interval [abstract]. Second Symposium on Lysosomal Storage Disorders; 2005 Mar 2-4; Athens
33. Banikazemi M, Ullman T, Desnick RJ. Gastrointestinal manifestations of Fabry disease: clinical response to enzyme replacement therapy. Mol Genet Metab 2005 Aug; 85 (4): 255-9
34. Guffon N, Fouilhoux A. Clinical benefit in Fabry patients given enzyme replacement therapy: a case series. J Inherit Metab Dis 2004; 27 (2): 221-7
35. Torregrosa V, de Arriba G, Checa D, et al. Health related quality of life in patients wtih Fabry disease treated with agalsidase beta: a twelve months follow-up study [abstract no. P34]. Fabry 5th Round Table Meeting; 2004 Oct 22-23; Warsaw
36. Dütsch M, Marthol H, Stemper B, et al. Small fiber dysfunction predominates in Fabry neuropathy. J Clin Neurophysiol 2002; 19 (6): 575-86
37. Cole A, Cousins A, Lee PJ. ERT infusion associated reactions in Fabry disease [abstract no. 338-P]. J Inherit Metab Dis 2004; 27 Suppl. 1: 172
38. Linthorst GE, Hollak CEM, Donker-Koopman WE, et al. Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta. Kidney Int 2004 Oct; 66 (4): 1589-95
39. Desnick RJ. Enzyme replacement therapy for Fabry disease: lessons from two α-galactosidase A orphan products and one FDA approval. Expert Opin Biol Ther 2004 Jul; 4 (7): 1167-76
40. Hughes DA, Ramaswam U, Elliott P, et al. Guidelines for the diagnosis and management of Anderson-Fabry disease [online]. Available from URL: http://www.dh.gov [Accessed 2005 Nov 21]
41. Barbey F, Hayoz D, Widmer U, et al. Efficacy of enzyme replacement therapy in Fabry disease. Curr Med Chem Cardiovasc Hematol Agents 2004 Oct; 2 (4): 277-86
42. Eng CM, Germain DP, Banikazemi M, et al. Fabry disease: guidelines for the evaluation and management of multi-organ system involvement. Genet Med 2006 Sep; 8 (9): 539-48
43. Warnock DG. Fabry disease: diagnosis and management, with emphasis on the renal manifestations. Curr Opin Nephrol Hypertens 2005; 14 (2): 87-95
44. Breunig F, Weidemann F, Beer M, et al. Fabry disease: diagnosis and treatment. Kidney Int Suppl 2003; 63 Suppl. 84: S181-5
45. Brenner BM, Grünfeld J-P. Renoprotection by enzyme replacement therapy. Curr Opin Nephrol Hypertens 2004; 13 (2): 231-41
46. Aaron JA, Ansani NT. Agalsidase beta: enzyme replacement therapy for Fabry disease. PT 2005 Apr; 30 (4): 222-4
47. European Medicines Agency. Replagal: summary of product characteristics [online]. Available from URL: http://www.emea.eu.int [Accessed 2005 Oct 5]
48. Schiffmann R, Kopp JB, Austin HA, et al. Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA 2001 Jun 6; 285 (21): 2743-9
49. Food and Drug Administration. Endocrinology and Metabolic Advisory Committee Meeting (14 January 2003) [online]. Available from URL: http://www.fda.gov [Accessed 2007 Feb 12]
50. Sakuraba H, Murata-Ohsawa M, Kawashima I, et al. Comparison of the effects of agalsidase alfa and agalsidase beta on cultured human Fabry fibroblasts and Fabry mice. J Hum Genet 2006; 51 (3): 180-8
51. Linthorst G, Hollak C, Ormel E, et al. A comparative trial of two enzyme preparations for Fabry disease [abstract]. European Study Group on Lysosomal Diseases Workshop; 2003 Sep 18-21; Podebrady
52. Wraith JE. The management of lysosomal disorders. Curr Paediatr 2004; 14 (5): 389-93
53. Breunig F, Wanner C. Enzyme replacement therapy for Fabry disease: proving the clinical benefit. Nephrol Dial Transplant 2003 Jan; 18: 7-9
54. Genzyme. Clinical studies of Fabrazyme [online]. Available from URL: http://www.fabrazyme.com [Accessed 2005 Oct 5]
55. Germain DP. The Fabry registry: natural history of renal disease and effects of enzyme replacement therapy [abstract no. 334-P]. J Inherit Metab Dis 2004; 27 Suppl. 1: 170
56. Cho ME, Kopp JB. Fabry disease in the era of enzyme replacement therapy: a renal perspective. Pediatr Nephrol 2004; 19 (6): 583-93
57. Mills K, Vellodi A, Morris P, et al. Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease. Eur J Pediatr 2004 Oct; 163 (10): 595-603
58. Young E, Mills K, Morris P, et al. Is globotriaosylceramide a useful biomarker in Fabry disease? Acta Paediatr 2005; 94 Suppl. 447: 51-4
59. Goi G, Massaccesi L, Burlina AP, et al. Lysosomal leukocyte β-D-glucuronidase during enzyme replacement therapy in Fabry disease. Biochim Biophys Acta 2005; 1741: 300-6
60. Utsumi K, Mitsuhashi F, Asahi K, et al. Enzyme replacement therapy for Fabry disease: morphologic and histochemical changes in the urinary sediments. Clin Chim Acta 2005; 360: 103-7
61. Mehta A. New developments in the management of Anderson-Fabry disease. QJM 2002; 95 (10): 647-53
62. Cole A, Cousins A, Morgan S, et al. Hospital, home or independence for agalsidase β infusions - the patients view [abstract no. P25]. Fabry 5th Round Table Meeting; 2004 Oct 22-23; Warsaw
63. Linthorst GE, Vedder AC, Ormel EE, et al. Home treatment for Fabry disease: practice guidelines based on 3 years experience in The Netherlands. Nephrol Dial Transplant 2006; 21: 355-60
64. Waldek S, Germain DP. Burden of Fabry disease in females and the importance of early therapy: an analysis of Fabry registry data [abstract no. 327-O]. J Inherit Metab Dis 2005; 28 Suppl. 1: 165
65. Peterschmitt MJ, Vázquez LA, Lago NR, et al. Enzyme replacement therapy (ERT) in a pediatric patient with Fabry disease (FD) [abstract no. P18]. Fabry 5th Round Table Meeting; 2004 Oct 22-23; Warsaw
66. Puccini R, Andreini B, Manni E, et al. Thirty months treatment experience with agalsidase β in a young patient affected by Fabry's disease [abstract no. P41]. 4th European Round Table on Fabry Disease; 2003 Oct 17-18; Munich